JS PD KB

Source Catalog

70 min read

Source Catalog

This generated catalog reflects every canonical by-photo Markdown file in data/processed/markdown/by-photo/ into the wiki layer. Each row links to the wiki source note, the topic-cluster section page that aggregates the source, and the canonical transcription.

#source notesectioncanonical transcriptionpagenav pathheadingsuncertainembedsrelated
120240722_181741gba-pd-asynmdPage 1 of 25Pipeline of GD & GBA-PDPipeline of GD & GBA-PD; NLRP3 PDE; 이주경; ADPD 2024; …301
220240722_181748gba-pd-asynmdPage 1 of 25Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; Two Group Proportion Comparison; GBA-PD / sPD / HC Biomarker Matrix; Cognitive Readouts; …402
320240722_181752gba-pd-asynmdPage 4 of 26Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; Eliglustat; Ambroxol; Mechanism; …622
420240722_181756gba-pd-asynmdPage 4 of 26Pipeline of GD & GBA-PD > Venglustat (Ibiglustat)Pipeline of GD & GBA-PD; Ambroxol; Clinical Trials Of Ambroxol; Venglustat (Ibiglustat); …722
520240722_181800gba-pd-asynmdPage 4 of 26Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; Venglustat (Ibiglustat); Clinical Trials Of Venglustat; LEAP Biomarker Notes; …842
620240722_181805gba-pd-asynmdPage 4 of 26Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; Prevail PR001; Preclinical; PR001 Single-Dose Efficacy Study In The Genetic Mouse Model; …852
720240722_181809gba-pd-asynmdPage 20 of 28Pipeline of GD & GBA-PD > Prevail PR001Pipeline of GD & GBA-PD; PR001 Mouse Data: 4L/PS-NA Genetic Mouse Model; PR001 Mouse Data: A53T + CBE Model; Dose-Finding NHP ICM Biodistribution; …942
820240722_181813gba-pd-asynmdPage 20 of 28Pipeline of GD & GBA-PD > SummaryPipeline of GD & GBA-PD; PR001A / PROPEL: Biomarker Goal And Interim Result; PR001A / PROVIDE; PR001A / PROGRESS; …322
920240722_181818gba-pd-asynmdPage 27 of 33Pipeline of GD & GBA-PD > TAK-341 (MEDI1341) > PK/PD modelPipeline of GD & GBA-PD; TAK-341 (MEDI1341); MEDI1341 Effector Function Note; MEDI1341 Affinity; …742
1020240722_181822gba-pd-asynmdPage 31 of 37TAK-341 (MEDI1341) > Clinical studiesPipeline of GD & GBA-PD; MEDI1341 MAD / TAK-341 POM Evidence; TAK-341 Preclinical Rat And NHP CSF a-Syn Evidence; Clinical Studies: SAD HV; …722
1120240722_181827gba-pd-asynmdPage 34 of 40aSyn programs > aSyn Propagation SuppressorPipeline of GD & GBA-PD; Program start as the point for formalizing alignment; aSyn Degrader; aSyn Propagation Suppressor; …702
1220240722_181831gba-pd-asynmdPage 34 of 40SNCA BTV (HDO) (PFR-4067-100) > Executive summary (Ryouta Maeda)Pipeline of GD & GBA-PD; SNCA BTV (HDO) Plan Table; DMPK MTV; Ryouta Maeda Notes; …1012
1320240722_181835gba-pd-asynmdPage 34 of 40SNCA ASO (WAVE) > Clinical plan of SNCA ASOPipeline of GD & GBA-PD; SNCA ASO PK/PD Relationship In Transgenic Mice; Clinical Plan Of SNCA ASO; PET SNCA ASO; …722
1420240722_181839gba-pd-asynmdPage 44 of 50TAK-071 > MechanismTAK-071 / Mechanism; Mechanism; TAK-071 PD Falls and cognition Timelines – Base Plan; Timeline of TAK-071; …1122
1520240722_181843cgas-cgampmdPage 44 of 50cGAS (cyclic GMP-AMP synthase, cGAMP synthase, =mb21d1) > SenescencecGAS: Senescence; cGAS Evidence Table Continuation; Paraquat / iPSC Correction Rows; Sliter 2018 Mouse / STING Deletion Table; …1112
1620240722_181847cgas-cgampmdPage 51 of 57cGAMP (cyclic GMP-AMP) > Assay development and monthly updatecGAMP Assay Development And Monthly Update; Partial Preceding cGAS Row; cGAS; cGAMP; …102
1720240722_181851parkinmdPage 51 of 57AGS (Aicardi-Goutieres Syndrome) / cGAS transition > Parkin > PARK2 Gene (=PRKN)AGS/ALS/cGAS to Parkin/PRKN Transition; AGS/cGAS Evidence Table Continuation; ALS with cGAS; Rationale; …312
1820240722_181855parkinmdPage 51 of 57Parkin > Parkin protein > KO ParkinParkin Protein; Concentration Of Parkin Protein; Commercial Parkin Product; ADDP In-Stock Parkin Proteins; …402
1920240722_181859parkinmdPage 62 of 68Parkin > Parkin protein > Location / States / Structure / Substrates transitionParkin Protein Location, States, Structure, Mutations, And Substrates Transition; Location Of Parkin Protein; States Of Parkin Protein; Parkin Activation State Diagram; …532
2020240722_181903parkinmdPage 66 of 71Parkin > Parkin protein > Substrates (& function) of Parkin > Parkin PDParkin Substrates, Animal Models, And Parkin-PD Prevalence; Substrates And Functions Of Parkin; Parkin PD - Prevalence Opening Note; Animal Models Of Parkin PD; …412
2120240722_181907parkinmdPage 66 of 71Parkin > Parkin PD > Clinical strategy > Natural Hx studyParkin-PD Prevalence And GE/GMS Biomarker Cohort Proposal; Prevalence Of Parkin Mutations; Young-Onset Combined Estimate; Late-Onset Estimate; …102
2220240722_181910parkinmdPage 71 of 77Parkin > Parkin PDGE/GMS Participant Mutation Table and MP-PD Parkin Variant Reanalysis; Preceding Application / Ethics Fragment; GE/GMS Participant Mutation Table; GE/GMS Participant Sample Matrix; …202
2320240722_181914parkinmdPage 71 of 77Parkin, > Parkin PDPRKN Variant Carrier Tables And Clinical Characteristics; PRKN Variant Table Continuation; N LoF + Deleterious Missense Carriers (AMP-PD); Page, 2020 #1751; …402
2420240722_181917parkinmdPage 71 of 77Parkin, > Parkin PDPRKN Clinical Characteristics And Pathogenic Mutation Evidence; Table 2. Demographic And Clinical Characteristics; PRKN Exon / Domain Variant Map; [Uncertain]ski, 2012 #692; …412
2520240722_181921parkinmdPage 71 of 77Parkin > Parkin PDPRKN Variant Annotation And Functional Assay Evidence; PRKN Variant Annotation Table, Continuation; Figure 1: Parkin Missense Variants Functional Alterations; Parkin Variant Functional Assay Table; …212
2620240722_181925parkinmdPage 71 of 77Parkin > Parkin PDPRKN Variant Classification Criteria And Portugal ARJP Mutation Summary; PRKN Variant Annotation Continuation; ACMG-AMP / Yi Pathogenic vs Benign Criteria; Summary For Clinical Trial From Yi; …1012
2720240722_181928parkinmdPage 71 of 77Parkin, > Parkin PDPortugal ARJP Table And Parkin Review Evidence; Table 1. Portugal ARJP Molecular Diagnosis; Klein, 2012 #915 Review; Akhit, 2016 #1061; …502
2820240722_181932parkinmdPage 88 of 94Parkin > Parkin PDKorean YOPD / PRKN ClinVar And OMIM Allelic Variant Notes; Korean YOPD / PARK2 Mutation Frequency Notes; ClinVar / OMIM PRKN Allelic Variants; Brain Net / ClinVarMiner / Structural Variant Notes901
2920240722_181936parkinmdPage 88 of 94Parkin > Parkin PDPRKN HGVS, dbSNP, And ClinVar Variant Tables; HGVS And dbSNP Variant List; ClinVar-Style PRKN Variant Rows601
3020240722_181939parkinmdPage 88 of 94Parkin > Parkin PDClinVar PRKN Variant Rows And Heterozygous PRKN Mutation Risk Notes; ClinVar-Style PRKN Pathogenic Variant Rows; Heterozygous Parkin Mutation1101
3120240722_181943parkinmdPage 98 of 104Parkin > Parkin PDPRKN Heterozygous Carrier Frequency, EOPD Risk, And Neuropathology; Heterozygous PRKN Carrier Notes; EOPD-Only Carrier Analysis; Population Estimates And Penetrance; …810
3220240722_181947parkinmdPage 98 of 104Parkin > Parkin PDPARKIN Autopsy Reports And PRKN-Related Clinical Phenotype Notes; Regional Neuronal Loss And Gliosis; Schneider 2017 PARKIN Autopsy Reports; Clinical Phenotype Notes; …911
3320240722_181950parkinmdPage 98 of 104Parkin > Parkin PDParkin PD Imaging Progression And Treatment Response; Carryover Notes Above The Imaging Section; Longitudinal Progression; Imaging Notes; …450
3420240722_181954parkinmdPage 107 of 113Parkin > Parkin PDParkin/PINK1 Mitophagy Evidence And pS65-Ub Detectability Notes; Parkin/PINK1 Mitophagy Evidence Matrix; Pathologic Row / Park2 Mutation; Extracellular Mitochondrial Expulsion Figure; …810
3520240722_181958parkinmdPage 107 of 113Parkin > Parkin PD > pS65-UbpS65-Ub Postmortem Evidence, NBB Comparison, And Assay Development Notes; Hou 2018 p-S65-Ub Aging And LBD Figures; Summary Table; Hou 2018 Reduced Or Absent p-S65-Ub Staining Figure; …441
3620240722_182001parkinmdPage 107 of 113Parkin > Parkin PDParkin Biomarker Strategy And Fibroblast Mitochondrial Evidence; Ubiquitin Linkage Basics; Biomarker Discovery And Validation Questions; pS65-Ub, Parkin Substrates, TE Biomarker, And Patient Selection; …1011
3720240722_182004parkinmdPage 107 of 113Parkin > Parkin PDParkin/PARK2 Fibroblast And iPSC Mitochondrial Evidence; Zilocchi 2020 PARK2 Fibroblast Continuation; Zilocchi Mitochondrial Proteomics And PhRET Proposal; Koentjoro Mitophagy And Rescue Notes; …131
3820240722_182007parkinmdPage 107 of 113Parkin > Parkin PDParkin Fibroblast Translation Plan And Parkin Delivery Evidence; Fibroblast, Lipid, And iPSC Evidence; Translation Plan Fibroblast And Leukocyte Complex Activity; Translation Plan Fibroblast; …1001
3920240722_182011parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100)PARKIN GT Expression Timeline And Parkin Delivery Evidence; Parkin Gene/Protein Delivery In Vivo Continuation; iCP-Parkin Model Notes; Bian MPTP Mouse And p62/SQSTM1 Notes; …401
4020240722_182014parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-…) > Executive summaryParkin GT Executive Summary, Clinical Strategy, And GBA PET Rationale; Parkin GT Development Timeline And Preclinical Plan; AAV9-Parkin Expression Comparisons; Vectors Carrying Codon-Optimized PRKN…; …312
4120240722_182018parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100) > Clinical strategyiCP-Parkin, USP30/NHP Plan, MitoCoP, And DBS+PRkn Strategy; GBA PET Rationale Carry-Over; GBA Expression Evaluation By Imaging; Competitor Table: iCP-Parkin And USP30/NHP Plan; …213
4220240722_182021parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100)PARKIN GT Decision Tree, Draft Timeline, Biomarker Matrix; Clinical Trial Endpoint Continuation; Decision Flow Notes; PARKIN GT Decision Tree; …221
4320240722_182025parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100)PARKN GT PRC1 Dataset, FXN Benchmark, In-Vivo Models, KOL Notes; Parkin KO Mice Biomarker Matrix Continuation; PRC1 In-Vivo Disease-Relevant / Function Model Dataset; Model Establishment In Parkin…; …101
4420240722_182029parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100)PARKIN GT Biomarker Plan, KOL Notes, And Natural History Meeting Notes; Prior Table Continuation And IP Filing; KOL Notes: Hattori, Michael Schlossmacher, Christein Klein; Hattori; …201
4520240722_182033parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100) > Natural Hx study > Sample size estimationMC1 PET / PRKN Natural History Study Proposal And Sample Size Estimation; MJFF / PPMI / Leuven MC1 PET Proposal Notes; Eberling / Timeline Fragment; Dated Notes; …221
4620240722_182036parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100) > Natural Hx study > Sample size estimation > NHP Studies >…Sample Size Estimation And NHP Studies Current Plan; Cohort / ClinGen Continuation; Sample Size Estimation; 90% Power / One-Sided p-value < 0.05; …221
4720240722_182040parkinmdPage 107 of 113Parkin > PARKN GT (Takeshi Hioki, PFR-4249-100) > NHP Studies > Current planNHP Studies Current Plan, CSF Sampling, And Parkin Correlation Evidence; NHP Studies: Current Plan; Study Columns / Timeline; AAV / Route / Criteria Notes; …002
4820240722_182043parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > NHP Studies > Bioanalytical method to differentiate human and monk…Bioanalytical Method To Differentiate Human And Monkey Parkin; Bioanalytical method to differentiate human and monkey Parkin,102
4920240722_182046parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > NHP Studies > Bioanalytical method to differentiate human and monk…Bioanalytical Method (continued) / NSRB / PRC Narrative / Natural Hx / Population Analysis / Quantitative BM Criteria; Post-PE; Population analysis; MJFF GGPD Project - Subjects; …202
5020240722_182050parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > Natural Hx study > Quantitative BM criteria / Target validationQuantitative BM Criteria, 20220119 / 20220130 Drafts, And Target Validation Plan; Quantitave BM criteria:; Target validation / Postmortem plan; Questions / Needs / criteria / timeline002
5120240722_182053parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > PE > PETTarget Validation (continued), PE I Planning, Parkin Binding Assay HTS History, And 20210709 11-Candidate List; Questions / Needs / criteria / timeline; PE I; Parkin Binding Assay / HTS history; …002
5220240722_182057parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > PE > PETParkin Binding Assay Development Log (20210615 → 20220715); 20211105 — ASMS results; 20211118; 20211217 — MAKOTO; …202
5320240722_182100parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > PE > PETParkin Binding Assay Termination Memo, Controversial Binders, And Start Of PE I Imaging GBA Plan; 20221215 — ADDP E000-25157 [parkin binding assay development]; 20230331_ADDP — ASMS assay developme…; …302
5420240722_182103gba-pd-asynmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > RD DDU’s PET Imaging GBA > PK/PD StrategyRDDU’s PE I Imaging GBA Plan (continued), PK/PD Strategy, 20201007 / 2021005 DMPK Meetings, AAV/PK Rationale, PRC1 Parkin Team Considerations, And [Benchmark] FA GT PRC1 PE Narrative; PK/PD Strateg…402
5520240722_182106parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > Safety > SharefolderScenario / Disease-Model Questions, Safety De-Risking Strategy, Sharefolder, Parkin Protein Notes, And Assays Table; Safety; Sharefolder; Timeline; …202
5620240722_182110parkinmdPage 144 of 150PARKN GT (Takeshi Hioki, PFR-4249-100) > TM > AssaysTeam Meetings / Update Meeting To Ceri Links, TM Assays Summary, NBB LLOQ Tables, Brain/CSF Comparison, pS65-Ub Reagents, Ubiquitin Linkage, IVC, And Start Of Parkin Activity Assay; Timeline; Resou…; …102
5720240722_182113parkinmdPage 144 of 150Parkin, > TM > Assays > IVC > Parkin activity assay? > TMQB > TPP/TCP > Unmet Needs > Vector production > P…Parkin; pS65-Ub assays; IVC; Parkin activity assay?; …400
5820240722_182116parkinmdPage 144 of 150PINK-1PINK-1; Protein; Phenotype; Pink1 mutation carriers; …702
5920240722_182120gba-pd-asynmdPage 144 of 150α-Synuclein and Lewy pathology in Parkinson’s disease > Supplement Positive vs Negativeα-Synuclein and Lewy pathology in Parkinson’s disease – Kalia and Kalia; GBA; Supplement: Positive vs Negative; Lysosomal function; …402
6020240722_182123gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary총정리 GBA protein & activity POSTMORTEM (continued); Synapse; 총정리 GBA protein & activity POSTMORTEM] Activity: hetero only 에선 kurzawa가 유일.; PD only / PD+ heteroz / Hetero only / GD only / PD+GD matrix; …502
6120240722_182126gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued)702
6220240722_182130gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued)502
6320240722_182133gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued)902
6420240722_182136gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Summary GBA protein & activity, GlcCer, Gl…Marker priority, 202107 Comp lysosomal target dashboard, and Summary GBA protein & activity, GlcCer, GlcSph; Marker priority; 202107 Comp lysosomal target dashboard; Summary GBA protein & activity,…302
6520240722_182139gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Summary GBA protein & activitySummary GBA protein & activity (continued), Summary table: GBA-PD patients, and Read the Paper (A) GBA Pathway Biomarkers; Summary table: GBA-PD patients; Read the Paper (A) (GBA Pathway Biomarkers…702
6620240722_182143gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > Read the PaperRead the Paper (>) GBA Pathway Biomarkers for Genetic and Idiopathic PD; Stratification by GBA pathway; 총정리중 GBA+PD에서 Lysosomal 반; S: Lysosome Lysosomal autophagy POSTMORTEM; …702
6720240722_182146gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity총정리 GBA protein & activity (continued), Sidransky 2012 review, Saposin C summary; [Gegg 2012] GBA activity / protein level; Table Below by Sidransky 2012 review; Saposin C summary; …602
6820240722_182149gba-pd-asynmdPage 144 of 150Pipeline of GD & GBA-PD > Summary > 총정리 GBA protein & activity > a-syn POSTMORTEMa-syn POSTMORTEM, a-syn SH-SY5Y + Cullen, a-syn PC only, FRACTIONATION; Choi 2011 요약; Postmortem rows (Mazzulli 2011, Murphy 2013, Murphy 2014); a-syn SH-SY5Y + Cullen: probably this is all for cel…; …402
6920240722_182153gba-pd-asynmdPage 197 of 204Pipeline of GD & GBA-PD > Summary > GBASidransky 2012 review (continued), PD-GD / GBA1-PD notes, GBA gene, GBA Mutation detection method; Sidransky 2012 review table continuation; PD-GD / GBA1-PD notes; GBA; …502
7020240722_182157gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > Structure of GBA proteinCarrier screening (continued), Structure / Function / Turnover / Expression / Lifecycle of GBA, activity (‘BGL test’); Carrier screening (continued); Structure of GBA protein (EC 3.2.1.45); Functio…; …002
7120240722_182200gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > GBA activity (BGL test)GBA activity (‘BGL test’); Product (kit)302
7220240722_182203gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > Catalytic pathwaySebastian table tail, Product (kit), Patient selection, Catalytic pathway, GSL flux, GluCer / GalCer isoforms, Gangliosides; Sebastian 20201124 (continued); Product (kit); Catalytic pathway; …102
7320240722_182207gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > Catalytic pathway > GangliosidesGluCer / GalCer isoforms (continued), Gangliosides, Sphingolipid pathway; GlcCer (Glucosylceramide); Gangliosides; Salvage pathway / Degradation; …202
7420240722_182210gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > Catalytic pathway > GangliosidesGangliosides Synthesis / Salvage / Degradation, Sphingolipid base, Disease cascade, Glycolipid metabolism, GBA chaperone; Gangliosides table (continuing from previous capture); Salvage pathway / De…; …502
7520240722_182213gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA > Catalytic pathway > Gangliosides > GlcSphDisease cascade tail, Glycolipid metabolism, GBA1 / GlcSph cycle, GBA chaperone / GCS inhibitor, GlcSph section; Ganglioside / sphingolipid degradation cascade tail (figure labels); 위 그림에서 cerebros…; …202
7620240722_182216gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Mutations SummaryGlcSph (continued) / GD: Dx, Epidemiology, Mutations Summary, mutation distribution; GD; Dx of GD; Epidemiology of GD (prevalence); …302
7720240722_182219gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Types and NeuropathologyPathophysiology In GD, GBA1 mutations, Event / Observational markers, Types and Neuropathology; Pathophysiology In GD; Types and Neuropathology202
7820240722_182224gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > GD 1 SummaryType 3 / GD3 (continued), CNS Sx in GD3, Parameters / GD 1 Summary; Type 3 (continued); Type 3 + PD; CNS Sx in GD3; …202
7920240722_182227gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > GD 3 SummaryGD 1 Summary tail, GD 3 Summary, GlcCer / GlcSph (detail), Narita 2016 patient table; GD 1 Summary (continued); SRT response (Smid 2016 #529); 2019 Lukina (Eliglustat); …502
8020240722_182230gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > GD 3 SummaryNarita 2016 patient body, Plasma in GD 1, Reference values, Animal models of GD; Narita 2016 #560 (continued); Plasma in GD 1; Experimental; …402
8120240722_182233gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Animal models of GD2019 Lukina (re-shown), Animal models of GD (continued), hGBA-L444P-tg8 mouse, GCB Mouse Model Substrate Comparison; 2019 Lukina Eliglustat (re-shown); Animal models of GD (continued); hGBA-L444P-t…; …102
8220240722_182237gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Animal models of GDD409V/D409V KI, GBA enzyme assay, conditional / nGD models, animal models comparison, Saposin C dose; D409V/D409V KI; GBA enzyme assay; MJFF: GBA D409VKi (hom) and crossed them with thy-1 aSyn or i…; …202
8320240722_182241gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Animal models of GD > Outcome measure in nGDStephens 1978 (continued), animal model literature rows, Outcome measure in nGD (SST / mSST); Stephens 1978 (continued); Animal model literature rows; Outcome measure in nGD; …202
8420240722_182245gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > Oculomotor measurementsOutcome measure (continued), Sample size in nGD, Oculomotor measurements, Saccade & PLR, Retina in nGD; Outcome measure (continued); Sample size estimation in nGD; Oculomotor measurements; …102
8520240722_182248gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > Retina in nGDHopf saccadometry tail, Retina in nGD / Retina & LSD, PLR / chromatic pupillometry, ERG, CRO / KOL, Lipofuscin; Hopf saccadometry (continued); Retina in nGD; Retina & LSD; …002
8620240722_182252gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GD > Parameters > Oculomotor measurementsRGC, Retinal PK, Short ciliary nerves, Antiepileptic drugs / retinal layers, RGC table, Study plan; RGC; Retinal PK; Short ciliary nerves; …102
8720240722_182255gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA-PD > Mutation GBA-PD2021 WORLDSymposium Ayuko Iverson, Narita Aya, Handover to Otake, GBA-PD penetrance, Mutation GBA-PD; 2021 WORLDSymposium Ayuko Iverson; Narita Aya (Iottori / Tottori univ); Handover to Otake on GD; …202
8820240722_182259gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA-PD > MOA GBA-PDPostmortem tail, Odds ratios, Phenotype / Cognition GBA-PD, Gene Dose Effect, MOA GBA-PD; Postmortem GBA-PD (continued); Odds ratios; Phenotype of GBA-PD; …202
8920240722_182303gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA-PD > Motor vs CognitionaSyn spreading tail, ER stress, OS, Inflammation, Motor vs Cognition, Cognition / Dementia; aSyn spreading (continued); ER stress; OS; …102
9020240722_182306gba-pd-asynmdPage 200 of 208Pipeline of GD & GBA-PD > GBA-PD > Animal models of GBA-PDIn vitro GBA-PD tail, 20191125 TAK Okai matrix, 20201209_ACi overview, Animal models of GBA-PD start; In vitro GBA-PD (continued); Below from 20191125 TAK Okai san slide gba-gt slide; Below from 20…; …002
9120240722_182310gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > Correction studiesAnimal models of GBA-PD tail, Correction studies (Rocha 2015 / AAV-A53T / Rockenstein 2016), S15 model, Sardi 2018 start; Animal models of GBA-PD (continued, A53T α-syn row); Correction studies; S1…; …102
9220240722_182313gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > Correction studiesCorrection studies tail, Message 모음 (GBA activity / GlcCer / GlcSph / a-syn), Goals, w reduced GBA activity Gene Therapy; Correction studies (continued); Message 모음 (GBA activity-GlcCer GlcSph - a-…; …102
9320240722_182316gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCAPatient Selection, nGD biomarker grid, Models / Omics, BMx Milestones, Patient recruitment, GBA vs UPDRS vs MOCA; Patient Selection; nGD biomarker / study-design grid; Model; …302
9420240722_182320gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCAGBA vs UPDRS vs MOCA (full literature comparison); Top longitudinal block (continued); Clinical dev Scenarios; genotype / Activity / Activity-DBS rows; …102
9520240722_182323gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > GBA vs UPDRS vs MOCACognition / Motor+cog / NfL tail, Progression summary, MRI GBA, Leocadi 2022, Thaler 2018, Agosta 2013; Progression summary; MRI GBA; longitudinal PPMI (Caminiti, 2022 #2706); …002
9620240722_182326gba-pd-asynmdPage 261 of 267Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBAMRI GBA tail (Caminiti DATSCAN / Leocadi / Thaler / Agosta / Filippi), PET Imaging GBA progress; MRI GBA (continued); DATSCAN longitudinal text; Leocadi 2022 #2708 (Italy); …002
9720240722_182330gba-pd-asynmdPage 276 of 282Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBAPET Imaging GBA history, Handover to Otake, Catalytic vs Allosteric binders, PET SC, GBA GT reporter table; Cf) Functional assay; History (Takeshi Wakabayashi / Otake / Makoto Fushimi); Handover to…; …102
9820240722_182333gba-pd-asynmdPage 276 of 282Pipeline of GD & GBA-PD > GBA-PD > PET Imaging GBA > GBA ActivatorAADC PET tail, Rationale of GBA PET, GBA expression evaluation, DDU/MJFF PET Imaging GBA, GBA Activator PRC1; AADC PET tail; Rationale of GBA PET; GBA expression evaluation by imaging; …202
9920240722_182336gba-pd-asynmdPage 276 of 282Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > RD-DDU GBA ActivatorGBA Activator criteria, Target profiles, Definition, GBA activity / GlcSph, RD-DDU GBA Activator, L444P Lyso-GL1, GBA GT start; GBA Activator criteria (continued); 20200428 Ceri meeting — Target pr…; …202
10020240722_182339gba-pd-asynmdPage 276 of 282Pipeline of GD & GBA-PD > GBA-PD > GBA GT > Executive summaryGBA GT Executive summary cassette selection, 3m-mice ICV screen, D409V 7-group selection, TE / PRC1 / Bettina, PD mouse model 4L/PS; STIG1 — cassette selection (criterion: GBA transgene expression…; …202
10120240722_182343gba-pd-asynmdPage 289 of 295Pipeline of GD & GBA-PD > GBA-PD > GBA GT > NHP studies > SNBR (Shin Nippon Biomedical Laboratories)In vitro GBA-PD POC, GBA-GT PK-PD strategy, Immunogenicity assays, NHP studies / SNBR; In vitro GBA-PD model (continued); GBA-GT [PK-PD strategy]; GBA GT [Immunogenicity assays] prc1; …002
10220240722_182346gba-pd-asynmdPage 289 of 295Pipeline of GD & GBA-PD > GBA-PD > GBA GT > TM GT GBA-PDSNBR/NHP tail, Otake-san’s update, RD-DDU GBA GT, TM GT GBA-PD planning matrix, GBA PET / mRNA / pathway; SNBR / NHP results (continued); Otake-san’s update; RD-DDU GBA GT; …002
10320240722_182351gba-pd-asynmdPage 295 of 301Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > NHP Translational ModelSample size estimation, Tx Paradigm, Biomarker Usage Map, DMPK PK/PD, Translational questions, Tempo code 43/6-100; Top header carry-over; Experimental Medicine; Tx Paradigm; …102
10420240722_182355gba-pd-asynmdPage 295 of 301Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > CompetitorGCB-BTV Executive summary tempo code, Biodistribution Studies, Comp Bio E-selectin, Competitor (Roche / BIAL LTI-291 / NCT05819359 ACTIVATE); GCB BTV Executive summary: tempo code 43/6-100; Biodist…; …102
10520240722_182358gba-pd-asynmdPage 295 of 301Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Competitor > Fusion ProteinBIAL LTI-291-004 tail, GBA activation table, Questions to BIAL, Gain Therapeutics GT-02287, Fusion Protein anti-TfR1 sdAbs; BIAL LTI-291-004; GBA activation table; Questions to BIAL; …002
10620240722_182402gba-pd-asynmdPage 295 of 301Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > RD DDU’s PET Imaging GBARD DDU’s PET Imaging GBA: GCB PET vs Lysosomotropic PET, BD study, Pharmacological modulation, Sample size estimation; PE1; GCB PET vs Lysosomotropic PET; Feasibility / BD study / Pharmacological m…; …002
10720240722_182406gba-pd-asynmdPage 307 of 312Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Acid ceramidase inhibitorIVC / TM / GBA2, Acid ceramidase inhibitor Yuta exec summary, Acid ceramidase / Farber lipogranulomatosis genetics; GBA2; Model system; Acid Ceramidase inhibitor — Executive summary (Yuta) (PFR-415…; …102
10820240722_182409gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > GALC (galactosylceramidase) > Genetics GALC for PDFarber lipogranulomatosis (continued), IPDGC ASAH1 LSD variants table, CADD C-Score note, GALC, Genetics GALC for PD; Farber lipogranulomatosis (continued); IPDGC LSD-Gene-Variants Supplementary Ta…; …202
10920240722_182412gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > GalactosylSph (=psychosine) > Assay of GalactosylSphGALC Genetics tail (NIH ClinVar tally), GalactosylSph (=psychosine, GalSph), Assay of GalactosylSph, Krabbe’s Disease start; NIH ClinVar Variation type / Clinical significance (GALC tail); Galactos…; …102
11020240722_182416gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Pipeline of KD > Target validation and correctionKrabbe psychosine continued, KD subtypes, Pipeline of KD, MOA for PD, Monthly Report (AC Inhibitor), Safety / Target validation; Krabbe psychosine continued; Pipeline of KD; MOA for PD; …102
11120240722_182419gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > GBA-PD > GBA Activator > Target validation and correction > 총정리중 GBA+PD에서 LysSafety BM tail, Kim 2018 carmofur GBA-KO results, Figure 7 / Figure 8, GalSph / psychosine summary, Krabbe questions, GALC enzyme summary; Safety BM tail; Target validation and correction — Kim 201…; …002
11220240722_182422gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > Supplement > CNS & PNS > Cerebral CortexAIM-PD-AD Emory study, Anatomy CNS & PNS, Classification of nervous system, Spinal nerve, Cerebral Cortex layers; AIM-PD-AD (Emory University); Preliminary Results [from 제목있는 문서]; [Plan]; …002
11320240722_182426gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > Supplement > Cerebral Cortex > Neuron > SynapsePNS / Spinal nerve / Cerebral Cortex (continued), Neuron / Synapse / Dendritic spines / PD-related changes; PNS / Somatic / Autonomic; Classification of nervous system; Spinal nerve; …102
11420240722_182429gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > Supplement > Neuron > Synapse > Postsynaptic density > Cranial nerveNeuron / Synapse continued, Asymmetric synapse density table, turnover, Synapse Asymmetric vs Symmetric, Postsynaptic density, Cranial nerve; Neuron; Common types of dendritic spines; Asymmetric sy…; …002
11520240722_182433gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > Supplement > Cranial nerve > Facial n > Nigrostriatal pathway > Basal GangliaCranial nerves nuclei tail, Facial n decomposition, Nigrostriatal pathway, Wakabayashi 2006 postmortem, SNc / DA neurons, Functional subdivisions of striatum, Basal Ganglia start; Cranial nerves nu…; …002
11620240722_182436gba-pd-asynmdPage 309 of 315Pipeline of GD & GBA-PD > Supplement > Basal Ganglia > MBN (nucleus basalis of M…) > Development of brain…Basal Ganglia tail, Substantia nigra MR sequences, MBN (nucleus basalis of Meynert), Development of brain, Brainstem (Pons / LDT / Locus coeruleus / Medulla), Borghammer 2021 BODY-FIRST vs BRAIN-FI…; …202
11720240722_182440gba-pd-asynmdPage 333 of 340Pipeline of GD & GBA-PD > Supplement > Gut > Commissures > Anatomy-Neurological signs > General(Borghammer, 2021 #1484) BODY-FIRST vs BRAIN-FIRST PD, Commissures, Anatomy-Neurological signs / General, motor anatomy table, Reported signs and symptoms; (Borghammer, 2021 #1484); Panel A — Conne…; …202
11820240722_182443gba-pd-asynmdPage 337 of 342Pipeline of GD & GBA-PD > Supplement > PD > Animal model > CROs > Database > Antibody > Epitope mappingPD neurological signs (motor + non-motor), Animal model / CROs / Database, Antibody monoclonal vs polyclonal, Epitope mapping, Affinity Maturation, APOE start; PD — Neurological signs; Animal model…; …002
11920240722_182447gba-pd-asynmdPage 340 of 345Pipeline of GD & GBA-PD > Supplement > Antibody > Epitope mapping > APOE > AQP4 potentiatorAQP4 potentiator (mislocalization / amyloid / p-tau), Glymphatic system in PD, AQP4 SNP, Burfeind 2017 cohort, Target validation, Ariba procurement, Astrocyte start; AQP4 potentiator; Increased amy…; …002
12020240722_182451gba-pd-asynmdPage 340 of 345Pipeline of GD & GBA-PD > Supplement > PET for astrocyte > aSyn > Animal models of aSyn > Non-Spreading ModelsReactive astrocytes A1/A2, (Li, 2019 #2281) molecular-expression table, PD and astrocyte, PET for astrocyte [11C]BU99008, aSyn animal models Non-Spreading + PD mouse models in QPS Line 61; Reactive…; …102
12120240722_182454gba-pd-asynmdPage 340 of 345Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Non-Spreading Models > Spreading ModelsNon-Spreading Models continued (WT Wade-Martins / F28tg / mjff / Mutant M83 PrP / KI rat / Truncated), Spreading Models, in vivo evidence, Atuka NHP results matrix; Animal models of aSyn — Non-Spre…; …102
12220240722_182457gba-pd-asynmdPage 340 of 345Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Spreading ModelsAtuka NHP Results matrix tail, Woodruff lab a-syn of mouse, PFF model (mouse pre-formed α-synuclein fibril), TAKEDA w Atuka Lentivirus, Gordon 2018 #587 / Chu 2019 / Luk 2012 / Duffy 2018 / Patters…; …002
12320240722_182501gba-pd-asynmdPage 351 of 357Pipeline of GD & GBA-PD > Supplement > aSyn > Animal models of aSyn > Non-Spreading Models > Spreading ModelsPatterson 2019 / SNCA BAC tg mouse / Bru 2013 #1370 row tail, spreading-models comparison table (PFF vs AAV with mechanism / Tx aims / Chung 2019 paragraph), Rey 2019 / 2018 / 2016 olfactory-bulb P…002
12420240722_182504gba-pd-asynmdPage 351 of 357Pipeline of GD & GBA-PD > Supplement > Non-Spreading Models > Spreading Models > Summary of a-syn animal mo…PFF preparation tail (storage / sonication / pilot validation), Vargas 2019 Summary of a-syn animal models Table 1, Transgenic vs Vector vs Injection matrix, MOA of aSyn spreading axon transport ta…; …002
12520240722_182507gba-pd-asynmdPage 351 of 357Pipeline of GD & GBA-PD > Supplement > Summary of a-syn animal models > MOA of aSyn spreading > Antibodies…MOA of aSyn spreading tail, Antibodies to aSyn (matrix + per-antibody host/Species/epitope), Vaikath 2019 #1599 antibody fragment map, Astrocytic aSyn (Aflaki 2020 GD1/GD2 iAstrocytes), CSF & Blood…; …002
12620240722_182510gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > MOA of aSyn spreading > Antibodies to aSyn > Astrocytic aSyn > CSF &…CSF & Blood (a-syn) — total aSyn levels, Why reduced in CSF? paragraph, King 2022 / Eusebi 2017 / Chou 2014 / Mollenhauer 2017 / Hall 2016 / Hansson 2014 / Compta 2015 / Majbour 2021 DenoPa / van S…102
12720240722_182514gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Astrocytic aSyn > CSF & Blood (a-syn) > Distribution > Expression of…CSF & Blood (a-syn) study rows continued (Schirinzi 2019 / Wang 2012 / PD01A / Lin 2019 / Chang 2019 / Wang 2020 / Chatterjee 2020 / Yamashita 2023 / Transpharmation Attyloid Blömeke 2022), Js summ…; …202
12820240722_182518gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Function (of normal aSyn) > Grading/Scoring of LB pathology > Homolo…Function (of normal aSyn), Grading/Scoring of LB pathology, Homology, In vitro studies of aSyn matrix, In vitro pharmacology assays; Function (of normal aSyn); Grading/Scoring of LB pathology; Homo…; …002
12920240722_182522gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aS… > Oligodendrocytic aSyn…Lewy Body / components / Two types / Toxic mechanisms / Lewy neurites, Lipid-aSyn interaction review, Mutations SNCA, Neurodegeneration and aSyn (Mori 2006 + Lee 2017 staging table), DBM, Oligodend…; …102
13020240722_182524gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn…PET aSyn vendor matrix (AC Immune / MODAG / University of Zurich / QST / Shenzhen / Merck / Mass Generic at Brigham) — Lead compound, Derivative, Structure, patient, Timeline, detects, in vitro Sat…002
13120240722_182528gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn…PET aSyn vendor matrix tail (Animal Cold/Hot, Animal in vivo PET, ARG, Preclinical Tox 2-rat studies, FH Protocol Eval, Protocol B-safety, FH F/U + team note, Four αSyn isomers, Alzforum half-life…002
13220240722_182532gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn…PET aSyn vendor matrix (Takeda CS 20230427, AlzForum FOSO28, MJF supported, Method comparison Smith 2023, SPAL-T-06 vs C05-05, Round 2 compound), Takeda co-owner license agreements; PET aSyn — vend…; …002
13320240722_182535gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Mutations SNCA > Neurodegeneration and aSyn > Oligodendrocytic aSyn…PET aSyn — QST Specified Clinical Trial planning (Operational lead, Funding MJF, Schedule, IND, Goal, Protocol, Process, Contract, Freedom to operate, GLP-Tox); QST Specified Clinical Trial — plann…002
13420240722_182539gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Neurodegeneration and aSyn > Oligodendrocytic aSyn > PET aSyn > Post…QST PET aSyn — process / contract / freedom to operate / GLP-Tox 20230808 plan, 20230714 Biomarker FlytoAs, Postmortem aSyn opener; GLP-Tox; (20230714) 3. Biomarker FlytoAs; Postmortem aSyn102
13520240722_182542gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Postmortem aSyn > Release > Safety for aSyn > KO of α-syn > Seed-Amp…Postmortem aSyn detailed staging table tail, Release of α-syn (sulfonylurea/SUR1 channel), Safety for aSyn / KO of α-syn (SNCA KO mice + Vilar 2018 + Markovich 2007), Seed-Amplification Assay (RT-Q…; …002
13620240722_182545gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Safety for aSyn > KO of α-syn > Seed-Amplification Assay > Quantific…Seed-Amplification Assay (RT-QuIC) — vendor / study rows (Russo 2020 / Onia 2021 / Pankha 2014 / Arnold 2021 / Brown 2023 / Brookmeyer 2021 / Olucium 2023 / Groves 2018), Quantification opener (Kin…; …002
13720240722_182549gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Seed-Amplification Assay > Quantification > Utility > Sources of aSynQuantification continued, end-point dilution + Calibration curve, application, Utility (Sidoroy 2023 Syn-target / RRK2-targeted / Aa-targeted), MJF workshop 20220421, Sources of aSyn (Cell/Brain/Re…; …002
13820240722_182552gba-pd-asynmdPage 359 of 365Pipeline of GD & GBA-PD > Supplement > Sources of aSyn > Skin aSyn > Species > Native conformation > Summar…Sources of aSyn detailed table (Recombinant / cell culture / Brain extract / Chemical synthetic with Method / pros / Product / parameter modelling rows), Skin aSyn (Syn-One test, Vasiliev 2018, Don…; …102
13920240722_182556gba-pd-asynmdPage 394 of 399Pipeline of GD & GBA-PD > Supplement > Summary aSyn species > NDE > Fragments (Truncation) > Assay TruncationPrimary aSyn species table tail, BDD update 202305 N-terminal α-synuclein, NDE (TAK-341 IDP / ADx + Cooveohe collaborations), Analyt-NIA ELISA SG4, Fragments / Splice variation (C-terminal truncati…; …002
14020240722_182559gba-pd-asynmdPage 394 of 399Pipeline of GD & GBA-PD > Supplement > Summary aSyn species > NDE > Fragments (Truncation) > Assay TruncationAssay Truncation continued (BDD update 202404, Kahn 2024 #2973 LC/MS isoforms in cingulate cortex), SNCA truncation isoforms (-126/-112/-98 with theoretical MW/aa), Bluhm 2021 #2157 schematic prote…; …002
14120240722_182602gba-pd-asynmdPage 394 of 399Pipeline of GD & GBA-PD > Supplement > NDE > Fragments (Truncation) > Assay Truncation > Structure of aSynHalia 2014 #2073 truncation table tail, Splice variation (4 αSyn transcripts SNCA-140/-126/-112/-98), Beyer 2008 Neurogenetics alignment, TAK-341 epitope on exon 5, Pathological Conversion + Pathol…; …002
14220240722_182606gba-pd-asynmdPage 394 of 399Pipeline of GD & GBA-PD > Supplement > Assay Truncation > Structure of aSyn > Synapse and aSyn > TM for aSy…Three major αsyn domains (N-terminal / NAC / C-terminal acidic tail), Synapse and aSyn (Calleri 2015 90% presynaptic deposits), TM for aSyn programs (Monomer / Pansson 2018 fibril CDMS, αSyn oligom…; …002
14320240722_182609gba-pd-asynmdPage 407 of 413Pipeline of GD & GBA-PD > Supplement > Synapse and aSyn > TM for aSyn programs > Turnover of aSyn > ATP13A2…TM for aSyn programs continued (TAK-341 in hippo / SNCA MEDI-1341 / SNCA p3 / How long?), Turnover of αSyn (Stefano 2018 #770 review with αSyn 240h half-life table by macroautophagy / cathepsin D /…; …002
14420240722_182613gba-pd-asynmdPage 407 of 413Pipeline of GD & GBA-PD > Supplement > ATP13A2 (Park9) > BBB / disruption / opener > BD > Process / BeyondB…ATP13A2 (Park9) MUA + Animal model + ATSURO MICRO + PXEAEAU Lysosomal protein degradation + SAL BAC + MPO score + Disruption of BBB + BBB disruption in PD + BBB opener Cordance + non-confidential C…; …002
14520240722_182617gba-pd-asynmdPage 407 of 413Pipeline of GD & GBA-PD > Supplement > Process > BeyondBio > Biobank > MJFPPMI Subjects/Available Data/Biospecimens, Shahn Rahmadhan + Por 20240228 Samantha notes, BioFiND, LRRK2 Cohort Consortium (LCC) Cross-sectional + Longitudinal + 23&Me Studies, S4 Systemic Syn Samp…; …002
14620240722_182620gba-pd-asynmdPage 407 of 413Pipeline of GD & GBA-PD > Supplement > BeyondBio > Biobank > MJF > Netherlands Brain BankNilo-PD continued (Cohort 1/2 + Available Data + Biospecimens), MJFF PPMI iPSC LINES, MJFF biobank contacts, 14 Types of Samples Available, Cohort Consortium ETBNRPP, tranSMART panel, Netherlands B…; …002
14720240722_182624gba-pd-asynmdPage 407 of 413Pipeline of GD & GBA-PD > Supplement > BeyondBio > Biobank > MJF > Netherlands Brain BankNetherlands Brain Bank tissue request continued (Sample transfer process / MTA / Transportation / cost per sample / IHC paraffin vs frozen / Molecular analysis / Location-Region-Analysis-Brain Amou…; …002
14820240722_182627gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > Biobank > MJF > Netherlands Brain Bank > Imperial College Brain BankBraak staging diagram tail (Thal 2002 + CERAD), Imperial College Brain Bank (Parkinson’s UK contacts + sample request + section types + tissue collections + materials + Jaewon QA), ~400 genotyped P…; …002
14920240722_182630gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > MJF > Netherlands Brain Bank > Imperial College Brain Bank > Other B…Imperial College Brain Bank PD genotyping table tail, sample availability + USDA statement, Imperial College Brain Bank Ariba PO process, Other Biobanks (NCNP J-PPMI Niigata univ TMM Cohort Study);…; …002
15020240722_182634gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > MJF > Netherlands Brain Bank > Imperial College Brain Bank > Other B…UK Brain Bank Network (NBB), BDR / NeuroChip / Camp dataset access table, NIH NeuroBiBank, NCRAD + EUFortuv MICAgou, PrecisionMed, BIOIVT hemoglobin, fibroblast vendors (NINOS / Cell Applications /…; …002
15120240722_182637gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > Netherlands Brain Bank > Imperial College Brain Bank > Other Biobank…Other Biobanks register continued (BIOIVT, vendors, Biochain, BioSEND, Novus Biologicals, ACCEGEN, Genomics England Banner Brain, NJ Brain Endowment Bank, NDRI, Neurobiobank Munich), Korean Open Go…; …002
15220240722_182641gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > B…Biobanks in Japan tail (Tsukuba1 + Kyoto-U Nagahama phase0), 20231009 Hooki Tsmurni Niigata univ, BWMVRINER NSTM BM Tracking (KPI) matrix, GBA Activity / GBM / GACer assay rows, CSF Sample (Sato) p…; …002
15320240722_182644gba-pd-asynmdPage 423 of 429Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > B…BIOMARKER tracker matrix continuation — lysosomal markers + aSyn / GlcSph / SCSF assay rows, Annapurna note, C8H CSF LCMS, Dr Margreith UMCS oligo aSyn discussion, MID/ELISA Parkin assay (cGAMP / p…; …002
15420240722_182704gba-pd-asynmdPage 440 of 447Pipeline of GD & GBA-PD > Supplement > Imperial College Brain Bank > Other Biobanks > Biobanks in Japan > B…BIOMARKER tracker continued — pSer65-Ub MJ assay (Atalee + SMC4Ps + Boston Bioscience LSBio antibody), MITOKININ NBB CSF, BoMM 240 NBB samples, NLM newsletter PhanoSwitch saliva-EV proteomics, Kore…; …002
15520240722_182708gba-pd-asynmdPage 440 of 447Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestraBIOMARKER tracker continued (Oxygen consumption + LC3 II), Stage / Date / Deliverables panel, BMx Platform / BPS / Feasibility / BFA / Technical / BTV / IVC / GPT / Tox / BFP stage matrix, BioOrche…; …002
15620240722_182711gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > BloodBioOrchestra ELAVL2 / miR-485-3p target table (continued), nELAVL bullet notes, Blood section opener (WBC / PBMC / plasma table, innate vs adaptive immunity, blood sample handling); ELAVL2 / miR-48…402
15720240722_182715gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > BloodBlood sample table tail (Suitable for RT PCR? / transcriptomics), Genomics England sample-handling guidance, Plasma / Serum / PBMC collection workflows, Lifespan / structure opener (classical monoc…; …302
15820240722_182718gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Bloo…Cell EM diagram citation, Lysosome / Example / 없는 것 table, mitochondria & aSyn notes, Sample preparation and process, Thawing and resting, Culture of PBMC, Vendors of PBMC table opening; Vendors of…202
15920240722_182722gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Braa…Vendors prospective tail, Braak Staging table (1-6 / HindBrain → Neocortex), Dx prodromal narrative, Zheng 2019 arrival-time chart, Masuda-Suzukake α-syn fibrils diagram, Takeda Braak / Propagation…; …102
16020240722_182725gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Braa…Burke 2008 H&Y vs Braak Stage scatter plot, Calcium channels (Cav subtypes table), SN DA neurons / Isradipine / In PD notes, askIT / CDA email thread (start); Fig. 3; Fig. 2; Calcium; …302
16120240722_182728gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…CDA email thread tail, CEI / Companies CEI / Vesalius distinct clinical feature types, Ambagon Argo molecular-glue 14-3-3 client table, Brain / CSF detection table, Giusto 2021 14-3-3 review, Under…202
16220240722_182732gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Underwood 2713 PFF tail, Zhao/Dzamko/muda LRRK2 14-3-3 notes, Ambagon-Takeda aSyn R&Rs confidential, 14-3-3 isoform/YWHA table, Deal concept / R&R, Mayor (AC immune) program, Competitor list, aSyn…202
16320240722_182735gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Companies CEI / Individual report (T3D, Neuracle, Nuravax, Escape Bio, Amsterdam Neuroscience, Muna Tx, GNS Healthcare, BlueRock, Genuv, Lysogene, Sunnybrook, Vincent / TMEM175, Annovis Bio, amimod…302
16420240722_182738gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Companies CEI / Individual report (continued): amimod hepatotox tail, ADRx tau, Nuravax follow-up, NeuroPn, Yumanity, Athira, Innomedica, Kyowa Kirin, BioVie NE3107, Logos Bio ADAD gene therapy / O…202
16520240722_182742gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Tx Nurr1:RXRa BRF110 program tail (TE / Neuroprotection / aSyn / neuroinflammation rows), Seelos / Herophilus / Myro Tx / AcureX / VesperBio / Priavoid / D&D Pharma (Neuraly), GLP-1R agonists clini…; …302
16620240722_182745gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Summary CEI continued: Confidential stage opportunities, Opportunities recently sent for triage, Relook at past MONITOR/Decline, NeuroD Landscape Refresh, NeuroD Heat Map, 20220906 Triage live disc…; …102
16720240722_182749gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Summary CEI continued: Key Projects Rundown / Heat Map / SE Core Team agenda recap, NeuroD SE Core Team Meeting (November 1, 2022) topics, JP Morgan Planning, Project Torchlight / Apex / Turing / B…; …202
16820240722_182752gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Summary CEI tail (Anima Biotech / Hi Julia), Heatmap CEI / Landscape / Meetings CEI sharepoint links, Cell lines (Neuronal catecholaminergic / Non-catecholaminergic / Non-neuronal tables), Cerevanc…; …202
16920240722_182755gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > CEI…Cerevance Pipeline (CVN424 / CVN766 / CVN293 / CD38 / KCNJ6 / GPR65), Current Portfolio Status: April 2022 timeline, Key worlds (evaluation / scientific confidence / Opportunity), Team table (funct…; …202
17020240722_182758gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Circ…Cerevance Team table (full), Centrifuge / ultracentrifuge note (Olga), Ceri Davies / Christine Klein meeting block, Mitochondrial biomarkers mini-table, Circuits / Direct pathway (cortex → striatum…; …102
17120240722_182802gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Circ…Healthy / Parkinson’s disease comparison panels, Direct pathway PD data table (SNc / D1 MSNs / GPi&SNR / thalamus / Cortex), Indirect pathway data table (D2 MSNs / GPe / STN / GPi&SNR / thalamus),…; …102
17220240722_182805gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose…Hypokinetic / Hyperkinetic Sx tail, Decision-making row, Clinical trial Examples of PD (SAD), MAD, Finding (study in the target population), Concomitant Medication, Dose finding / Dose ranging stud…; …202
17320240722_182808gba-pd-asynmdPage 446 of 453Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose…C-Path SWEDD vs DAT-Deficient slide (Conrado 2018), Summary from below percentages, Description table (intervention-based suppression of disease progression), WATCH-PD sample-size narrative, 2017 S…302
17420240722_182811gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dose…GBA GT v2_GP sample-size tail, 20190423 Sclareol-NS imaging strategy 4-panel chart, Sample size calculation (proportion / Visions Pharmaceutical 2021 #1755 / Lundkya 2013), Innovative design / Inte…302
17520240722_182815gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…AV133 sample-size tail, Cognition / Cholinergic / Anatomy (basal forebrain BF corticopetal projection / PPN-LDTC / MVN / striatal cholinergic interneurons), Pathway PET enzyme table (ChAT → VAChT →…; …302
17620240722_182818gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Cumulative prevalence summary slide (50-70% / 25% bullets), vMRI literature matrix (Burton / Ramirez-Ruiz / Xia / Melzer / Goldsby / Apostolova / Mak studies); CUMULATIVE PREVALENCE OF COGNITIVE AN…202
17720240722_182821gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…vMRI tail (Segura 2018 cortical thickness), Aβ NPT brain/CSF, dual-syndrome hypothesis (fronto-striatal vs cholinergic posterior), Imaging (Aβ in PD / Forrencys 2018 / Florencys 2016 postmortem), s…; …302
17820240722_182824gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Pathology tail (basal forebrain / hippocampal cholinergic system / Frontal cortex / ChAT activity), Hellinger 2008 review postmortem note, Consortium (MOBILIZE-PD / Verily / Takeda Japan / Watch /…; …202
17920240722_182827gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…PPMI Study Documentation / Operations Manuals overlap, Other PPMI Program Protocols, Data and Biospecimen Use Agreements and Policies, LBD (Aria 2020 #2728) sub-study, Innovative Medicines Initiati…; …102
18020240722_182830gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…MJFF Grant Portal block (Pre-Proposals / Coordinating Lead PI / Full Proposal Invitations), MJF imaging meeting along with MJFF, WORKSHOP 202111 day2 conference slide, Canada / PDGENeration / Steph…; …102
18120240722_182833gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Rare Disease Data Registry tail / Dr. Takeda AMED tail, Contacts (extended), MTM Contact info, Cortellis Drug Discovery Intelligence (Sequence search BlastP/BlastN), CROs (오시리스 사 / KBio Health / AT…; …202
18220240722_182836gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…CSF Flow / Blood contamination / Fluid compartments / Barriers in the brain; Blood contamination; CSF Flow; Fluid compartments in the brain; …002
18320240722_182839gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Brain CSF illustrations / Barriers table overlap, Brain structure (ECF / parenchyma / ventricular system), CSF turnover (4×/day), SE as a Surrogate for Assessing CNS Exposure (Lamotrigine / Phenyto…; …302
18420240722_182842gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Mouse CSF / Rat CSF serial sampling tables, Cytokines Classification (Interleukins / Chemokines / Interferons / TNF / Colony Stimulating / TGFβ), Interleukins (IL-1β / IL-6 / IL-9 NLRP3 narrative),…; …302
18520240722_182845gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Interferons / ISG overlap, Multiplex cytokine assay (R&D Systems Luminex 50uL / Proteome Profiler Rat XL / Olink 48 mouse panel / 96 mouse exploratory / 92 human / pricing / detectability), DBS sec…002
18620240722_182848gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Olink panels overlap, D&D Phamatech (NLY01 NCT04154072), DBS (EARLYSTIM Schuepbach 2013 + Indication / Side effects / Prognosis bullets), DDC (Aromatic L-amino acid decarboxylase): AADC enzyme / Ke…; …002
18720240722_182851gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…DDC AADC overlap (Cieselska 2017 / 18F-L-DOPA / Why increase / Qs), Diagnosis of PD overlap, Stages of early PD overlap, Digital section opener (Lipsmeier 2018 PMID 29701258 / 6-month Phase 1b smar…; …102
18820240722_182854gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…APDM ML / Lipsmeier 2022 / WATCH-PD overlap, APDM walking-feature characteristic (Tremor / Bradykinesia / Postural / Mobility / Speech), Timed walking / Stand and go / Postural sway / standing walk…; …002
18920240722_182857gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Adams 2022 supplemental table tail, NCT06219629 LEARNS observational basket study, TAK071-2002 crossover trial, TAK994-2001 PK/safety phase 2 trial, TAK-341 IMED1341 MAD Apple Watch feasibility, NE…002
19020240722_182900gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…WATCH-PD/Konektm overlap, SWO-001 Japan / CarefoOne Apple Watch ongoing rows, (Drowy, 2020 #1223) Comparison of drug pharmacotherapy survey table, Digital PD Summary (US/EU vs Japan studies), Disea…102
19120240722_182904gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…Disease area biomarker table tail (CSF p65-Ub / Functional mito CSF BM / imaging / α-syn / Lysosome rows), Omics resources (Silberring 2010 #1148), DJ-1 (Park7) block, DLB vs PDD vs MSA comparison…; …102
19220240722_182907gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…DLB vs PDD vs MSA imaging / pathology rows (VMAT PET / sMRI Burton-Borroni-Cohn / aSyn PET DWI Pellecchia / Synuclein-only inclusions Olivera 2021 / Aβ load Hepp 2018 / NFT / Brainstem-Limbic-neoco…002
19320240722_182910gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Chol…DLB vs PDD vs MSA tail (Aβ / NFT / GWAS / GBA / SNCA / CSF αsyn / Clinical trial), MSA agent registry (Lithium / Stem cell / Mesenex αSyn immunotx / Bicheron verepinstat), DNA damage section opener…; …002
19420240722_182913gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…DNA repair tail (Homology-directed / Non-homologous / Microhomology-mediated / Direct reversal / Translesion synthesis), DNA damage in PD (Sariguchi 2021 #1782 γH3AX / DNA glycosylase upregulation…; …102
19520240722_182916gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…Dopamine metabolites in PD — CSF / blood / urine correlation rows (LeWitt 1992 / Stefani 2017 / Wichi 2021), Anderson 2017 DA-HVA L-DOPA narrative, Dormant dopaminergic neurons (Heo 2020 astrocytic…002
19620240722_182919gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…Microdialysis tail (Wegrzynowicz 2019), Nigrosomes (dopaminergic) neurons in SNpc (postmortem 98% loss in nigrosome-1), brain MRI swallow-tail panels, Dopa Imaging — dopamine synthesis capacity (11…002
19720240722_182922gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…DAT imaging products (PET / SPECT tracers / sensitivity / normal reference values), DATscan analogue references, DATscan in PD findings (caudate / putamen / striatum percent reduction; UPDRS correl…; …102
19820240722_182926gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…Datscan progression in PD (Simuni 2018 #2272 Table 5 baseline-Month 6/12/24/48; Table 7s Year 1/Year 4 percent change), Arthur volcano AD-PD database mining longitudinal DaT trajectories + power an…; …202
19920240722_182929gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…TABLE 4C / 4A correlation tables (Year 0/1/2/4 Spearman MDS-UPDRS × DATSCAN SBR), 6Mo vs UPDRS III / 4Y vs UPDRS III longitudinal rows (Tribais 2019 / Toiraj 2022), Correlation between Datscan & hi…; …102
20020240722_182932gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…Rodent imaging centers overlap, KBigD studies (KH Park 2020 18F-PR-PE2I MPTP-mouse / Js 2019 11C-PE2I 6-OHDA-rat / AAV SNCA-rat), Charles Rivers studies, SWEDD section (Marek 2018 #1283 PD PROTOpos…; …002
20120240722_182935gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…Neuromelanin pigment biochemistry (Zecca review caption: NM-pigment biosynthesis & NM-containing organelle formation in human SN), Function (chelate iron / pigmented neurons vs TH correlation, Mor…; …002
20220240722_182938gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Dopa…NM-MRI validation table tail (Cassidy / Isaias / Ito), Longitudinal NM-MRI in PD (Cristian Salinas 20220625 commentary; Wirth 2022 #2071 Saljas cohort 140+97; vsNMc / disNMc / vsNMs annualized deca…002
20320240722_182941gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Drug…79 early PD vs 32 HV cohort tail (cleaner copy at 20240722_182938), Dopamine Receptor Imaging continued (Booth 2015 #1745 raclopride PET / Bezard 2001 #1748 MPSP Monkey ABL plots), Druggability sec…002
20420240722_182944gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Drug…Druggability; DWI VS DTI302
20520240722_182947gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Drug…Druggability (continued tail); DWI VS DTI; Dyskinesia; Clinical presentation of Dyskinesia in PD; …202
20620240722_182950gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Drug…Dyskinesia; Clinical presentation of Dyskinesia in PD; MOA of Dyskinesia; Outcome Measures Dyskinesia; …302
20720240722_182953gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Drug…Pipeline of Dyskinesia (continued); EEG; Bands; EEG in PD202
20820240722_182956gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG…EEG in PD (continued); Limitations; Sleep EEG; Effect size302
20920240722_182959gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG…Sleep EEG (continued); Effect size (continued); post-hoc; NLRP3 (or ASC) vs TH; …502
21020240722_183002gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > EEG…Correlation / Exact test (continued); Efficacy modelling; Electrophysiology; Anatomy of a neuron; …102
21120240722_183006gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Elec…Electrophysiology; Anatomy of a neuron; Methods; Resting potential (Inactive state); …202
21220240722_183009gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Elec…Resting potential (Inactive state); Process; voltage-dependent ion channels; ERB; …102
21320240722_183012gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Endo…Endoplasmic Reticulum (ER); Exosome; function; Limitation of exosome; …302
21420240722_183015gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Olig…Oligodendrocyte-derived exosome (continued); Eye; Fibroblast; Cell model system for CNS?; …302
21520240722_183018gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GABA…GABA (continued); Further readings; GABA imaging; Gait; …302
21620240722_183021gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Glad…Gladstone Institute; Glutamate Imaging; Goals; FY22; …401
21720240722_183024gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Goal…GWAS; Modifiers; Summary502
21820240722_183028gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GWAS…LSD genes and variants in the IPDGC cohort402
21920240722_183031gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GWAS…LSD genes and variants in the IPDGC cohort; GPR37; Target; Glossary; …402
22020240722_183034gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > GPR3…GPR37; Target; Glossary; H&Y (Hoehn and Yahr)402
22120240722_183037gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > H&Y…HA-tag; Hearing loss; Nigrosome1 MRI; Nigrosome1 MRI202
22220240722_183040gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Hear…Heuron; Hirozane, Yoshihiko; HR Performance Review (including 360); Human Biological Samples302
22320240722_183044gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > HR P…Human Biological Samples; Imaging; PET; Immunity402
22420240722_183047gba-pd-asynmdPage 498 of 504Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Immu…Immunity; Autoimmunity (Autoantibody); Evidence; ImmunoPET; …202
22520240722_183050gba-pd-asynmdPage 626 of 632Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Infl…aSyn (therapeutic) antibody; Comorbidity; Summary (sento M)502
22620240722_183055gba-pd-asynmdPage 626 of 632Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Como…Signals; Inflammasome; In vitro model of neuroinflammation202
22720240722_183058gba-pd-asynmdPage 626 of 632Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Infl…Imaging neuroinflammation302
22820240722_183101gba-pd-asynmdPage 626 of 632Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Infl…302
22920240722_183104inflammationmdPage 626 of 632Imaging neuroinflammationImaging neuroinflammation; DPA714; 3rd Generation TSPO402
23020240722_183108inflammationmdPage 626 of 632Imaging neuroinflammation > DPA714 > 3rd Generation TSPO3rd Generation TSPO302
23120240722_183111clinical-pdmdPage 626 of 632Diagnosis of PDCNS-Resident Macrophages (CRM); Microglia; Structure of microglia; Phenotypes of microglia401
23220240722_183115gba-pd-asynmdPage 626 of 632Pipeline of GD & GBA-PD > Supplement > Other Biobanks > Biobanks in Japan > BIOMARKER > BioOrchestra > Infl…Markers of microglia; Priming of MG; Receptors on microglia302
23320240722_183118inflammationmdPage 626 of 632Pyroptosis MOAPipeline of Inflammation; Pyroptosis MOA; low CSHL (cold spring harbor) 2020 Abstract253 MOA; ptosis evidence/MOA/Correction in PD501
23420240722_183121inflammationmdPage 626 of 632Pipeline of Inflammation > Pyroptosis MOA > Pyroptosis evidence/MOA/Correction in PDPyroptosis evidence/MOA/Correction in PD302
23520240722_183125inflammationmdPage 651 of 660Pipeline of Inflammation > Pyroptosis MOACorrection501
23620240722_183128inflammationmdPage 651 of 660Pipeline of Inflammation > Pyroptosis MOA > Pyroptosis evidence/MOA402
23720240722_183132inflammationmdPage 661 of 670NLRP3 > Structure-1β; IL-1 Receptors; Toxicity; NLRP3 Structure502
23820240722_183135gba-pd-asynmdPage 661 of 670Inflammation > NLRP3 > Neuronal > Caspase 1 > aSyn splicing > Catalytic cleftExpression; GWAS; Neuronal; KO; …301
23920240722_183138inflammationmdPage 661 of 670Cryopyrin-Associated Periodic Syndromes (CAPS)CAPS; Questions; Classification; Genetics; …302
24020240722_183141inflammationmdPage 661 of 670Pipeline of Inflammation > CAPS > WBC > Dx > ARP > SxWBC; Dx; f. Dx criteria; ARP; …402
24120240722_183144inflammationmdPage 661 of 670NLRP3 > ToxicityTx402
24220240722_183147inflammationmdPage 661 of 670Inflammation > NLRP3 > Tx502
24320240722_183150inflammationmdPage 661 of 670Pipeline CAPSLimitations; Outcome measures; Registry; Animal model; …402
24420240722_183153inflammationmdPage 661 of 670Pipeline of Inflammation > Pipeline CAPS > Animal model > TM > NLRP3 inhibitor (Marianthi) (PFR-4231-100)NLRP3 inhibitor (Marianthi) (PFR-4231-100); Executive summary502
24520240722_183156inflammationmdPage 661 of 670NLRP3 inhibitor (Marianthi) (P…) > AD > Assays of pyroptosisAD; In vivo; NBB (project number: 1513); Assays of pyroptosis402
24620240722_183159inflammationmdPage 661 of 670Inflammation > NLRP3 inhibitor (Marianthi) > AD > Assays of pyroptosisAssays of pyroptosis302
24720240722_183203inflammationmdPage 695 of 701Assays of pyroptosis502
24820240722_183206inflammationmdPage 695 of 701Pipeline of Inflammation > Pipeline CAPS > NLRP3 inhibitor (Marianthi) (PFR-4231-100) > AD > Assays of pyro…Quanterix:; Progress402
24920240722_183208inflammationmdPage 698 of 704Assays of pyroptosis > Budget & FTE > Ceri > ChemistryCBID (BST):; Summary of NLRP3 biomarker detection methods in the literature; Budget & FTE; Ceri; …402
25020240722_183212inflammationmdPage 698 of 704Comp BioClinical; Combining MBMs Multi-Modal Biomarkers and Biomarker Panels; Comp Bio; PD; …501
25120240722_183215inflammationmdPage 698 of 704Assays of pyroptosis (cytokine / chemokine omics) > ADInterleukins; AD; Overall502
25220240722_183218inflammationmdPage 698 of 704Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > AD > Chemistry > Clinical > Comp Bio > CompetitorAD; Molecular subtypes AD; Competitor402
25320240722_183222inflammationmdNLRP3 inhibitor (Marianthi) (P…) > Pipeline / Competitors502
25420240722_183225inflammationmdPage 713 of 719Inflammation > Competitor > HavrdaCompetitor; Havrda; Clinical; Subpopulation strategy; …201
25520240722_183228inflammationmdPage 713 of 719HavrdaTamoxifen-inducible Nlrp3 allele.; HED; IHC; In Vivo strategy402
25620240722_183231inflammationmdPage 713 of 719Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > In Vivo strategy > Havrda > HED > IHCCompounds; In vivo strategy details; Cell numbers in AAV1/2 + pSyn mice402
25720240722_183234inflammationmdPage 713 of 719In Vivo strategy > KatyBiomarkers measurements for planned in vivo studies; Subchronic; Katy602
25820240722_183238inflammationmdPage 726 of 732Inflammation > In Vivo strategy > KatyAtuka aSyn / BzATP rat dose grid; 098: 2ND ATUKA mouse, 10 mpk, (vs AAV+veh) TH ↓ Putamen / NBB / Imperial402
25920240722_183241inflammationmdPage 726 of 732In Vivo strategy > Katy > LongitudinalWorkflow; Longitudinal502
26020240722_183244inflammationmdPage 733 of 738Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > Katy > Longitudinal > Metabolomics > Microglial Im…Metabolomics; Microglial Imaging402
26120240722_183247microglia-imagingmdPage 735 of 741Microglial ImagingStructure302
26220240722_183250microglia-imagingmdPage 735 of 741Microglial Imaging > Modelling > NHP > PDEModelling; NHP; PDE; Meeting with PRC office over PDE-rough notes101
26320240722_183252pet-imagingmdPage 735 of 741PE > PET > Chemistry PETPE items; Schedule; PET; Chemistry PET; …502
26420240722_183255inflammationmdPage 744 of 750Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > Microglial Imaging > NHP > PDE > PE > PETSummary502
26520240722_183258pet-imagingmdPage 747 of 753PETGOF mouse; 6OHDA Rat; LPS (systemic) Rat502
26620240722_183301pet-imagingmdPage 747 of 753NHP > PDE > PE > PETLPS (bain injection) Rat; Other models; ARG Postmortem-AD; PET activities (with start/end date); …101
26720240722_183305pk-gt-pharmacologymdPage 747 of 753PK/PD > PK/PD StrategyPK/PD; PK/PD Strategy; MOUSE Free Brain Exposure; HUMAN Free Brain Target; …502
26820240722_183308inflammationmdPage 747 of 753Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > PK/PD > PK/PD Strategy > Postmortem > ProteinaPablo Pelegrin, Roche (2 times, biofluid); Proteina Bob. Scienist.com: 48D6FD: Assay development: NLRP-INFLMMASOME402
26920240722_183311operationsmdPage 759 of 765Reactome > SafetyReactome; Safety; General; Non-GLP Tox studies402
27020240722_183314operationsmdPage 759 of 765Postmortem > Proteina > Reactome > SafetyAccumulation Ratio; CVB: Reactive metabolites; CVB Activities; Ocular; …201
27120240722_183318operationsmdPage 765 of 772Safety > PhospholipidosisTR06693098-related findings indicating generalized phospholipidosis; Toxicokinetics parameters for TR0663098 (mean, n=3); Tissue concentrations of TR06693098 (mean, n=3); [Clinical testing for DIPL]; …502
27220240722_183321inflammationmdPage 765 of 776Pipeline of Inflammation > NLRP3 inhibitor (Marianthi) > Reactome > Safety > Screening flow > ShareFolderEstablished NLRP3 Inhibitor Screening Flow; ShareFolder402
27320240722_183324other-mechanismsmdPage 765 of 776TAU > TCP > TimelineSleep; TAU; TCP; Timeline; …402
27420240722_183327biomarkers-outcomesmdPage 775 of 782Transfer > Team > Translational questions > RBD+PDTranslational questions; RBD+PD; NCNP CSF sample; Email: RE: RBDの研究・期間延長させていただけますでしょうか_263-5 — Kamiguchi, Hidenori401
27520240722_183332inflammationmdPage 779 of 784Stratification strategy on neuroinflammation > RNA-Seq (Daria) > C5aR1 (Woodruff, Univ of Queensland)Stratification strategy on neuroinflammation; Postmortem study; RNA-Seq (Daria); C5aR1 (Woodruff, Univ of Queensland)402
27620240722_183335inflammationmdPage 782 of 789Pipeline of Inflammation > Complement / C5aR1 > MOA > Woodruff’s study plan > Screening > DDWoodruff’s study plan with C5aR1 inhibitors; Screening; DD402
27720240722_183339operationsmdPage 782 of 789DD > ProgressProgress; GBA; team:; Takeda plan for PMX318 evaluation under MTA with UQ; …402
27820240722_183342inflammationmdPage 788 of 796Background > Pipeline C5aR1 > Innomedica (Haven) > In Vivo ClibrationBackground; Pipeline C5aR1; Innomedica (Haven); In Vivo Clibration301
27920240722_183345parkinmdPage 792 of 798GAPFREE3 (iPSC, Yuya Kunisada) > Mid-brain organoids with FTD-tau (University of Cambridge) > Iron > Normal…GAPFREE3; SA’s group; Mid-brain organoids with FTD-tau; Iron; …402
28020240722_183348inflammationmdPage 792 of 798Pipeline of Inflammation > Postmortem > Imaging of Iron > IT > JayaPostmortem; Imaging of Iron; IT; Jaya; …402
28120240722_183351inflammationmdPage 798 of 805Pyroptosis evidence/MOA…FOX-1: Marios politis:; K-BIOX; KOLs501
28220240722_183354inflammationmdPage 798 of 805Minee Choi > Kv1.3 > Kyoko Yoshikawa > Lee JonghunMinee Choi; Kv1.3; Pipeline [Muna Tx]; Kyoko Yoshikawa; …201
28320240722_183358samples-collaborationsmdPage 805 of 811Liquid Nitrogen > LongitudinalLiquid Nitrogen; Longitudinal502
28420240722_183401lrrk2mdPage 811 of 817Longitudinal > 4 LRRK2 > Animal models of LRRK2 > Inflammation and LRRK24 LRRK2; Animal models of LRRK2; Inflammation and LRRK2302
28520240722_183404genetics-pathwaymdPage 811 of 817MutationInflammation and LRRK2; MOA; Mitophagy and LRRK2; Mutation; …400
28620240722_183407lrrk2mdPage 814 of 820Pipeline of LRRK2 > Summary (Schneider…) > DNL201 (=GNE-7…) > Clinical trialsPenetrance; Pathology; Phenotype; Clinical progression; …301
28720240722_183410lrrk2mdPage 814 of 820DNL151 (=BIIB12…) > Clinical trials > PreclinicalPreclinical; Clinical trials; Preclinical (BIIB094)302
28820240722_183413lrrk2mdPage 814 of 820LRRK2 Clinical trials > Other LRRK2 pipeline > Prevalence > Protein > Lysosome > Structure of Lysosome > Io…Clinical trials; Other LRRK2 pipeline; Prevalence; Protein; …302
28920240722_183416lysosome-autophagymdPage 826 of 832Lysosomal Enzymes⊞PhReT; BMs; RD-DDU; Lysosomal changes in PD; …500
29020240722_183419lysosome-autophagymdPage 826 of 833RD-DDU > Lysosomal changes in PD > Lysosomal Enzymes > Assessment of lysosome > MarkerAssessment of lysosome; Process401
29120240722_183423lysosome-autophagymdPage 829 of 835Macroautophagy > Microautophagy > CMA > BMP (Bis(monoacylglycero…))Macroautophagy; Autophagy markers; Microautophagy; CMA; …402
29220240722_183426mitochondriamdPage 829 of 835Magnetoencephalography (MEG) > Maiko Tanaka > Metabolomics > MIBGComp Bio; Lysosomal Targets; Turnover; Machine Learning; …502
29320240722_183429operationsmdPage 829 of 835Milestones (process)600
29420240722_183432mitochondriamdPage 829 of 835Maiko Tanaka > Metabolomics > MIBG > Milestones (process)Translational and early clinical research plans to (Portfolio Entry — PE); Portfolio Entry (PE); Candidate Selection (CS); High-level scientific definition/requirements for each sub-stage in NS DDU…; …301
29520240722_183436gba-pd-asynmdPage 829 of 835ShareFolder > Mitochondria > Astrocyte > asyn and mitochondria > Evidence in sPDMitochondria; Astrocyte; asyn and mitochondria; Evidence in sPD402
29620240722_183439mitochondriamdPage 844 of 851Function > Steps of cellular respirationFunction; Steps of cellular respiration502
29720240722_183442mitochondriamdPage 851 of 857Structure of mitochondriaLocation of mitochondria; Structure of mitochondria; MITOCHONDRIAL RESPIRATORY CHAIN: COMPOSITION600
29820240722_183446mitochondriamdPage 851 of 860Mitochondrial DNA home… > Location of mitochondria > Structure of mitochondria > RotenoneMitochondrial Respiratory Complexes (I-V); Rotenone-binding site / Common MC1 inhibitors (pesticides)-binding sites / The catalytic core sites of MC1; Rotenone; a- rotenone; …401
29920240722_183449mitochondriamdPage 855 of 862Structure of mitochondria > Mitochondrial respiratory chain complexes > Subunits of complex I > MAM (Mitoch…Table 1. Subunits of complex I.; MAM (Mitochondria-associated membranes); Imaging of MAM402
30020240722_183452mitochondriamdPage 855 of 862Mitochondrial DNA > Mitochondrial genes > Methylation of mt DNA > Mitochondrial turnover > Mitochondrial tr…Mitochondrial DNA; Mitochondrial genes; Methylation of mt DNA; Mitochondrial turnover; …402
30120240722_183455mitochondriamdPage 855 of 862SummaryAssessment of mitochondria; Summary600
30220240722_183458mitochondriamdPage 855 of 862Mitochondrial Regulators > Circulating cell-free mitoch… > Assessment of mitochond… > SummaryMMP (Mitochondrial Membrane Potential, ΔΨm); Mito (Circulating cell-free mitochondria); [Mitochondrial integrity]; [MT in exosome]; …301
30320240722_183501mitochondriamdPage 870 of 873Mitochondrial Regulators > Circulating cell-free mitochondrial DNA > Assessment of mitochondrial function >…502
30420240722_183505mitochondriamdPage 870 of 876Assessment of mitochond… > Summary > 31P MRS imaging > PrincipleMRS Imaging; Principle402
30520240722_183508mitochondriamdPage 873 of 879Studies of 31P MRSStudies of 31P MRS; Principle600
30620240722_183511mitochondriamdPage 873 of 879Summary > 31P MRS imaging > Principle > Studies of 31P MRSHuman (Prasuhn, 2019 #1475) (Prasuhn, 2020 #1476); PME; Cons; Christen Klein clinical trials; …401
30720240722_183514gba-pd-asynmdPage 882 of 888Studies of 31P MRS > MELAS (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) > MJFFMELAS (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes); MJFF; Pipeline C5aR1402
30820240722_183518gba-pd-asynmdPage 882 of 889Studies of 31P MRS > Oliver Bandmann, MD,… > MELAS (Mitochondrial enc…) > MJFF402
30920240722_183521inflammationmdPage 882 of 889Stratification strategy on…Stratification strategy on Mitochondria500
31020240722_183524mitochondriamdPage 888 of 895Genes Mitochondria > PRS Mitochondria > MC1 PET > MC1 PET studiesProtein; [Targeted approach]; Genes Mitochondria; PRS Mitochondria; …401
31120240722_183528mitochondriamdPage 891 of 898USA’s group402
31220240722_184103parkinmdPage 897 of 920Preclinical support > Fibroblasts > PARK2 cellsPreclinical support; Fibroblasts502
31320240722_184130parkinmdPage 900 of 922Preclinical supportBudget; Sample preparation examples; Shipping; Sample preparation original plan; …600
31420240722_184135mitochondriamdPage 900 of 922PRS Mitochondria > MC1 PET > MC1 PET studies > Preclinical supportFuture direction; Update 20211210 (Yoshirou, only living cell); Update 20220108 (1st small repeat); Planning saturation analysis (PROTEIN amount 400 ug/well); …502
31520240722_184138parkinmdPage 903 of 922GAPFREE3 > Preclinical supportSaturation analysis-only HC (GM01650); Concentration response curve (only HC (GM01650)); Saturation analysis (Main)301
31620240722_184142parkinmdPage 910 of 922GAPFREE3 > Preclinical support > ARGARG402
31720240722_184146mitochondriamdPage 913 of 923MOA of MitophagyMC1 PET in FA; Microdialysis; Mitophagy; MOA of Mitophagy; …500
31820240722_184149mitochondriamdPage 916 of 926mitophagy in vivo… > Monthly update > MRI > Classificationmitophagy in vivo; Monthly update; GBA-PD GT; BMx Milestones project timeline; …401
31920240722_184153pet-imagingmdPage 919 of 9297T MRIHyperintensity (=T2 hyperintensity); DBM (Deformation-Based Morphometry,); 7T MRI; sequence301
32020240722_184156gba-pd-asynmdPage 922 of 933MSA > aSyn in MSA > CSF aSyn in MSAMRI in PD; MRS in PD; MSA; Animal models of MSA; …402
32120240722_184200gba-pd-asynmdPage 925 of 935Brain BankOligomeric aSyn in MSA; RT-QuIC in MSA; RBC aSyn in MSA; Skin aSyn in MSA; …600
32220240722_184203gba-pd-asynmdPage 925 of 937Brain Bank > Clinical Progression > Clinical Trial example > CohortBrain Bank; Clinical Progression; Clinical Trial example; Cohort; …401
32320240722_184206msamdPage 928 of 939MSADiagnosis of MSA – three levels101
32420240722_184210msamdPage 934 of 943MSA > Dx of MSA > ImagingDx of MSA; Criteria for the Diagnosis of Probable MSA; Criteria for Possible MSA; Genetics; …302
32520240722_184213biomarkers-outcomesmdPage 934 of 945NFLDTI; Potential MRI endpoints to assess drug effects; DWI; MRS; …600
32620240722_184216msamdPage 937 of 947MSA > Outcome Measures > Unified MSA Rating Scale (UMSARS)NfL in MSA, Outcome Measures, UMSARS, MSA-QoL Start; NfL Longitudinal Table (continued); NfL Cross-Sectional / Longitudinal CSF and Blood Comparison; Outcome Measures; …502
32720240722_184219msamdPage 940 of 949MSA > Outcome Measures > PathologyOutcome Measures; Multiple System Atrophy Health-Related Quality of Life (MSA-QoL); ICLEMSA (Items That Change Largely in Early-Stage Multiple System Atrophy); Table 4 — The eight items with the la…; …202
32820240722_184223msamdPage 946 of 957MSA > Pipeline MSA > Disease-modifyingPipeline MSA; Disease-modifying; Verdiperstat – Phase 2: NCT02388295201
32920240722_184226msamdPage 949 of 959MSA > Pipeline MSA > Disease-modifying402
33020240722_184230msamdPage 949 of 960Pipeline MSA > SubtypePipeline MSA — Disease-modifying / Symptomatic / Subtype; Key Ongoing Trials for MSA: Clinical Development Timelines; Symptomatic; Subtype; …500
33120240722_184233msamdPage 952 of 963MSA > Pathology > TM in MSA > Mucolipidosis (ML)Pathology — comparison across MSA subtypes; TM in MSA; Mucolipidosis (ML)402
33220240722_184237samples-collaborationsmdPage 958 of 967NDU > DDU Subteam > 20201022 Kick off > SharefolderMultiple Sclerosis; Natalia Boukharov; NDU, DDU Subteam; 20201022 Kick off; …302
33320240722_184240operationsmdPage 958 of 969Sharefolder > NeurodegenerationClinical subteam; Quarterly Feedback; Neurodegeneration502
33420240722_184244samples-collaborationsmdPage 964 of 972Neurogenomics PartnershipNeurogenomics Partnership; Neuro Genomics Partnership 2020-2021; Meeting 20220727 @Clotilde Degroot, Ms.:; Niemann-Pick disease (partially visible at bottom edge)400
33520240722_184247lysosome-autophagymdPage 964 of 974Niemann-Pick diseaseNiemann-Pick disease502
33620240722_184250biomarkers-outcomesmdPage 964 of 975MOA of NPC > NFL > Age > PDMOA of NPC; NfL; Age; PD201
33720240722_184253biomarkers-outcomesmdPage 974 of 980NFL > PD[20230404 JS Datscan and CSF NFL effect size]; Turnover402
33820240722_184256operationsmdPage 974 of 981NHP > Marmoset > Cynomolgus (Macaca fascicularis) > On-Off PhenomenonNHP / Marmoset / Cynomolgus / On-Off Phenomenon; Structure; NHP; Marmoset Translation; …400
33920240722_184300biomarkers-outcomesmdPage 981 of 988Outcome measures > Original UPDRS (1987)Organoids; Outcome measures; Original UPDRS (1987); MDS-UPDRS’ = Modified UPDRS (2008)500
34020240722_184303biomarkers-outcomesmdPage 984 of 990Outcome measures > Original UPDRS (1987) > MDS-UPDRS’ = Modified… > (Simuni, 2018 #198)Outcome measures — MDS-UPDRS; TABLE 2. MDS-UPDRS total scores over time in treated and untreated PD patients401
34120240722_184306biomarkers-outcomesmdPage 984 of 992MoCA (Montreal Cognitive Assessment)Milestone-based approach; Takeda UPDRS analysis; MoCA (Montreal Cognitive Assessment)302
34220240722_184310inflammationmdPage 984 of 994Stratification strategy on… > Pathogenicity of variantSleep; Ataxia; SARA (Scale for the Assessment and Rating of Ataxia); Neuropsychiatric Inventory (NPI); …300
34320240722_184314genetics-pathwaymdPage 992 of 999Pathogenicity of variantTable 3 Criteria for Classifying Pathogenic Variants; Table 4 Criteria for Classifying Benign Variants; Table 5 Rules for Combining Criteria to Classify Seque…; …400
34420240722_184317genetics-pathwaymdPage 995 of 1003PADD > Pathogenicity of variant > Pathway Analysis > Example of Pathway AnalysisPathway Analysis — comparison; Example of Pathway Analysis601
34520240722_184320genetics-pathwaymdPage 998 of 1005Pathway Analysis > Example of Pathway Analysis > PDE301
34620240722_184324gba-pd-asynmdPage 1002 of 1008PDE > PIAS2 > Pipeline of PD > aSyn - AntibodyPIAS2; Pipeline of PD; aSyn - Antibody; α-syn sequence targeted by mAbs and vaccines400
34720240722_184327gba-pd-asynmdPage 1002 of 1008Pipeline of PD > aSyn - Antibody > aSyn - Small moleculesPasadena trial design (Part 1 / Part 2 / Part 3); mAbs summary; aSyn - Small molecules400
34820240722_184330gba-pd-asynmdPage 1008 of 1017Pipeline of PD > aSyn - VaccineaSyn - Vaccine — NPT200-11 row tail (Neuropore), Anle138b / Ionis / PD1601 row tail, AC Immune PD01A (ACI-7104) and PD03A AFFiRiS row group (P2 VacSYn / NCT06015841, P1 MSA / NCT02270489, P1 NCT015…; …131
34920240722_184333clinical-pdmdPage 1008 of 1018Pipeline of PD > GTaSyn - Vaccine / GT Transition; SAD Study - PK Data To Date / MEDI1341; PD01A / Long-Term Follow-Up Notes; PD01A / GBA-PD Row; …721
35020240722_184337clinical-pdmdPage 1018 of 1024Pipeline of PDPipeline of PD — gene therapy and cell therapy (continued); Cell therapy; Inflammation; 기타 MOA들400
35120240722_184340other-mechanismsmdPage 1018 of 1026기타 MOA들201
35220240722_184344biomarkers-outcomesmdPage 1025 of 1030GT > Cell therapy > Inflammation > 기타 MOA들400
35320240722_184348other-mechanismsmdPage 1025 of 1034기타 MOA들200
35420240722_184351clinical-pdmdPage 1025 of 1034Pipeline of PD > 기타 MOA들기타 MOA들 (continued)500
35520240722_184354other-mechanismsmdPage 1033 of 1040기타 MOA들301
35620240722_184357inflammationmdPage 1036 of 1044Inflammation > 기타 MOA들 > Pittsburgh University > Greenamyre LabPittsburgh University; Greenamyre Lab; INTERESTS; Burton Lab; …300
35720240722_184401samples-collaborationsmdPage 1036 of 1046Pittsburgh University > Burton LabBurton Lab; Chu Laboratory; Hastings Laboratory; Postmortem; …202
35820240722_184404clinical-pdmdPage 1042 of 1050Prodromal PD > Definition of Prodromal PD > MDS research criteria for prodromal PDPrevalence of PD; Prodromal PD; Prodromal PD studies; Definition of Prodromal PD; …400
35920240722_184407clinical-pdmdPage 1042 of 1051Takeda efforts in prodromal PDPhenoconversion; Takeda efforts in prodromal PD; Clinical trials in prodromal PD; P2P Platform Trial301
36020240722_184410samples-collaborationsmdPage 1045 of 1054P2P Platform Trial > Progranulin (PGRN) > Denali Progranulin PTV > PD and PGRNProgranulin (PGRN); Denali Progranulin PTV: PGRN (DNL593); Biomarkers of DNL593; Mode of action model; …500
36120240722_184413gba-pd-asynmdPage 1048 of 1056PD and PGRN > MJF > Prophylactic vs Therapeutic > Protein tagsMJF; Prophylactic vs Therapeutic; Protein tags; PRS; …302
36220240722_184417molecular-biologymdPage 1051 of 1060PD ProteomicsPD Proteomics (continued); Stastics; Power analysis400
36320240722_184420pet-imagingmdPage 1057 of 1066Terminology > Psychosis in PD > pipeline > RadiochemicalPsychosis in PD; pipeline; Radiochemical; Rare Disease101
36420240722_184424pet-imagingmdPage 1060 of 1071Pipeline > Radiochemical > Rare Disease > PETRare Disease Prevalence, Lysosomotropic / LAMP1 Binder, Postmortem Lysosomal Evidence; Rare-Disease / Prevalence Table; Lysosomotropic vs Lamp1 Binder; Postmortem Human Lysosomal Evidence in GBA-PD; …502
36520240722_184427pet-imagingmdPage 1060 of 1071PET > RBD (REM sleep behavior disorder) > Symptom > IncidencePSYCHIATRIC OUTCOMES IN PD; CUMULATIVE PREVALENCE OF COGNITIVE AND PSYCHIATRIC DISORDERS OR TREATMENT IN PD; PARKINSON DISEASE VS. HEALTHY CONTROLS OVER TIME; PD CHARACTERISTICS CONT.; …400
36620240722_184430msamdPage 1063 of 1076Pipeline MSA > Disease-modifying > RBD screening question… > RBDQ-HQ (REM sleep…) > Mechanism > Patholo…Dx; Screening tools of RBD; RBD screening questionnaire (RBDSQ); RBDSQ-J; …302
36720240722_184434biomarkers-outcomesmdPage 1066 of 1078RBDGBA → RBD; GBA → RBD → PD; RBD → PD; RBD in PD; …400
36820240722_184437gba-pd-asynmdPage 1072 of 1082RBD PDD/DLB > GBA pathway in RBD > NCNP collaboration for RBD > Reference range (normal range)RBD PDD/DLB / NCNP Collaboration / Reference Range; NCNP collaboration for RBD (& PD) (Yuji); NCNP, Dr. Yuji Takahashi — Molecular BM discovery using CSF and PBMC from PD and RBD patients; Referenc…; …400
36920240722_184440biomarkers-outcomesmdPage 1072 of 1084Retina > Issues Retina > Rob RubensRetina / Issues Retina / Rob Rubens; Issues Retina; Retina: a window of brain; Rob Rubens; …400
37020240722_184443pk-gt-pharmacologymdPage 1075 of 1085Measurement of OS > Route of Administration > ICM (= Suboccipital puncture) > Peripheral leak/transductionEvidence in PD; Measurement of OS; Route of Administration; ICM (= Suboccipital puncture); …301
37120240722_184447clinical-pdmdPage 1079 of 1088Pipeline of PD > GTAAV biodistribution evidence, ICM delivery comparison, and start of intra-parenchymal slide; NHP AAV9 biodistribution evidence (top of capture); Administration Via Intra-cisterna magna (ICM) Allows…; …101
37220240722_184450biomarkers-outcomesmdPage 1085 of 1095Outcome measures > Sandy > Screenig > Scientst.com > SchizophreniaHLI 20210806; Sandy; Screenig; Scientst.com; …402
37320240722_184453samples-collaborationsmdPage 1085 of 1097SECONDMENTSECONDMENT500
37420240722_184456biomarkers-outcomesmdPage 1088 of 1100MoCA (Montreal Cognitive…SEPT14; [Share]Folder400
37520240722_184459operationsmdPage 1094 of 1104SharefolderInternal Takeda Resource Link Table (continues)201
37620240722_184503biomarkers-outcomesmdPage 1094 of 1106SCOPA-AUT > Shipment > Sigma-1 Receptor (σ1R) > σ1R PET > SILKShipment; Sigma-1 Receptor (σ1R); σ1R PET; SILK; …502
37720240722_184506biomarkers-outcomesmdPage 1097 of 1109biomarkersbiomarkers; Skin Biopsy; Sleep / Outcome measures; Prevalence; …200
37820240722_184509genetics-pathwaymdPage 1105 of 1113Example of Pathway Analy…Grouping subjects into several equal groups; Method to stratify: ]500
37920240722_184513biomarkers-outcomesmdPage 1106 of 1116Succinate > Surrogate endpoint > Symptom > SynapseSuccinate / Surrogate endpoint / Symptom / Synapse; Succinate; Surrogate endpoint; Symptom; …300
38020240722_184516clinical-pdmdPage 1106 of 1118Pipeline of PD > GTSV Steps, Genetics Synapse, Imaging Synapse, and start of Readouts of Synapse; SV Steps; 3-1) clathrin-mediated pathway (major pathway); 3-2) kiss and run pathway; …400
38120240722_184519biomarkers-outcomesmdPage 1112 of 1122Synapse > Neuronal pentraxins > Synaptic change in PD > Target Identification > TDP43 > TMEM106B > TMEM175Synapse / Neuronal pentraxins / TDP43 / TMEM106B / TMEM175; Functional; Neuronal pentraxins; Synaptic change in PD; …500
38220240722_184522samples-collaborationsmdPage 1112 of 1123P2P > TMQB > Toxins > Paraquat > MPTPEffect size; MOA; Pipeline; TMQB; …402
38320240722_184525samples-collaborationsmdPage 1118 of 1130P2PP2P; Pesticides; Effect on Inflammation; 6-OHDA (=Oxidopamine); …300
38420240722_184529samples-collaborationsmdPage 1121 of 1131P2P Platform Trial6-OHDA (=Oxidopamine); MPTP and 6-OHDA Down-regulate Parkin Activity And/or Its Expression — Boosting Cell Death Signaling; MPTP Model; 6-OHDA Model; …400
38520240722_184532molecular-biologymdPage 1124 of 1135Proteomics > Issues and SolvingTranscranial sonography tail and Transcriptomics in PD; Transcranial sonography in PD; Transcriptomics in PD; Overview of transcriptomics in PD; …001
38620240722_184535mitochondriamdPage 1124 of 1137TRAP1 > MOATRAP1 — MOA And Pipeline Page (Page 1124); Bulk / Single Cell Transcriptomics Plan (continuation); Midbrain DA neurons; TRAP1; …402
38720240722_184539other-mechanismsmdPage 1130 of 1140(no nav path)TREM2 Agonists, Tremor, and TRPML1 Notes600
38820240722_184542lysosome-autophagymdPage 1133 of 1145TRPML1TRPML1; Tottori univ collaboration GD; Catecholamine pathway400
38920240722_184545biomarkers-outcomesmdPage 1133 of 1145RBDQ-HO (REM sleep…Established PD Therapy; Experimental PD Therapy; Striatum / Substantia nigra schematic; Corticostriatal Glutamatergic Afferent / Striatum schematic300
39020240722_184550clinical-pdmdPage 1137 of 1149Symptom > Tx pattern WWAnimal model treatment table tail, Korean Rx market, Tx pattern WW/Korea, Turning, and Types; Animal model (table tail); Korean PD drug list; Tx pattern WW; …101
39120240722_184553other-mechanismsmdPage 1140 of 1155Unmet Needs > UPS > MOA of UPSUK Biobank Top Differentially Expressed Proteins, Unmet Needs, And Start Of MOA Of UPS; UK Biobank — Top differentially expressed and decreased proteins for G20 Parkinson’s Disease Case-Control ana…402
39220240722_184557biomarkers-outcomesmdPage 1147 of 1157Reference range (normal range)Reference range (normal range); UPS impairment in PD patients; Ubiquitin; Gene Ubiquitin; …300
39320240722_184600biomarkers-outcomesmdPage 1147 of 1161Rob RubensResource: Mizushima 2020; Urine; Yan; Figure S5 panels; …400
39420240722_184604pet-imagingmdPage 1147 of 1162VMAT Vesicular monoamine transporter-2 > VMAT PET > [18F]AV-133AAV gene-therapy trial table tail, Yuhan section, VMAT2 PET ([18F]AV-133), and sample-size analysis; Animal model / clinical trial table tail (continues); Yuhan 유한; VMAT Vesicular monoamine transpo…; …202
39520240722_184607molecular-biologymdPage 1154 of 1167[MOLECULAR BIOLOGY] > Central Dogma ProcessNeurodegeneration In The Nigrostriatal Dopamine System (DaTscan / VMAT2 PET) And Central Dogma Process (Page 1154); Neurodegeneration in the nigrostriatal dopamine system — Parkinson’s disease (Tak…; …502
39620240722_184610molecular-biologymdPage 1161 of 1171[MOLECULAR BIOLOGY] > DNA > cDNA / ASO / DNA structure[MOLECULAR BIOLOGY] / DNA; ASO; HDO Structure; DNA; …400
39720240722_184613biomarkers-outcomesmdPage 1164 of 1173SILKSILK; Three major purpose of DNA QC; Mutations; SNP Database400
39820240722_184616operationsmdPage 1164 of 1175Species difference{Richards, 2015 #1508}; Population, Disease-Specific, and Sequence Databases; Nomenclature of mutations and sequence variations; Recommendations for the description of sequence variants; …300
39920240722_184620molecular-biologymdPage 1168 of 1178General recommendations > Detailed recommendations > Protein level > GeneralHGVS Sequence Variant Nomenclature — General Recommendations (continues); Detailed recommendations; Protein level; General300
40020240722_184623molecular-biologymdPage 1171 of 1182Insertions > Deletion/insertions (indels) > ExtensionsHGVS Protein Nomenclature — Silent Changes, Substitutions, Deletions, Duplications, Insertions, Indels, Extensions (Page 1171); Silent changes; Substitutions; Deletions; …402
40120240722_184626biomarkers-outcomesmdPage 1177 of 1186Synaptic change in PDSynaptic change in PD; More changes in one individual; fathmm prediction pathogenic score; Genotyping Methods; …400
40220240722_184629genetics-pathwaymdPage 1177 of 1188(no nav path)Genetic Testing400
40320240722_184633genetics-pathwaymdPage 1180 of 1192Genetic Association StudySNP (Single Nucleotide Polymorphism); Definition; GATK (Genome Analysis Toolkit); Copy number variation (CNV); …302
40420240722_184636genetics-pathwaymdPage 1180 of 1195polygenic risk score > Steps of PRS STUDY > Results of PRS > eQTLStatistical analysis tail, Genetic support level, Genetic prevalence workflow, and PRS; Statistical analysis (table tail); Genetic support Level; Genetic support level; …401
40520240722_184639genetics-pathwaymdPage 1187 of 11974 LPSKey points about eQTLs critical for target discovery efforts; trans-eQTL Investigation; eQTL Investigation of Human Spinal Cord Tissue; pLoF (predicted loss-of-function); …402
40620240722_184643molecular-biologymdPage 1191 of 1200[MOLECULAR BIOLOGY] > Mutations > Genotyping MethodsCre-LoxP recombination; TET EXPRESSION SYSTEM; (A) Present: GWAS / Future: Trans-OWAS; Transcription / mRNA / Structure301
40720240722_184646molecular-biologymdPage 1200 of 1205mRNA > miRNA > RNA binding proteins (RBPs) > Transcriptome > Database of transcriptome > Gene Expression An…miRNA / RBPs / Transcriptome; RNA binding proteins (RBPs); Transcriptome; Gene Expression Analysis (Transcriptome); …300
40820240722_184649molecular-biologymdPage 1203 of 1208Spatial transcriptomics > Single cell transcriptomics > Gene ontology > [Protein] > Structure of protein >…Spatial / Single Cell Transcriptomics, Gene Ontology, Protein; Spatial transcriptomics; Single cell transcriptomics; Experimental Steps; …402
40920240722_184653molecular-biologymdPage 1206 of 1212ProteomicsMethods of proteomics; Database of Protein structure; GFP (green fluorescent protein); Homology402
41020240722_184656molecular-biologymdPage 1209 of 1215Protein-Protein Interaction (PPI) > Post-Translational ModificationsProtein-Protein Interaction (PPI) / Post-Translational Modifications; Protein-Protein Interaction (PPI); Types of PPI; Assays of PPI; …400
41120240722_184700other-mechanismsmdPage 1209 of 1215UPS > Metabolomics > Omics > Cis- vs Trans- > Intracellular deliveryUPS impairment tail, PTM wheel, Metabolomics/Omics, Cis-vs-Trans, Intracellular delivery; UPS impairment in PD patients (table tail); Frequency; PTM wheel — Target Protein; …100
41220240722_184703operationsmdPage 1216 of 1221Assay Development LC-MSMouse strain; Transgenic Mouse; Fluorescent proteins; Process; …302
41320240722_184706molecular-biologymdPage 1216 of 1222[Protein] > Assays > LC-MS vs ELISA > Flow cytometry > FRET > ELISA > Sandwich ELISA > SIMOA > MSDLC-MS vs ELISA / Flow Cytometry / FRET / ELISA / Sandwich ELISA / SIMOA; Products; LC-MS; LC-MS vs ELISA; …402
41420240722_184710molecular-biologymdPage 1222 of 1234[Protein] > Assays > ELISA > AlphaLISA > PCR > SPR > DNA Synthesis assays > Cell Signaling Assay > Binding…ELISA / AlphaLISA / PCR / SPR / DNA Synthesis / Cell Signaling Assays; ELISA vs WB; AlphaLISA; PCR; …402
41520240722_184713molecular-biologymdPage 1225 of 1235[MOLECULAR BIOLOGY] > Mutations > Genotyping MethodsSAR (Structure Activity Relationships); Immunoprecipitation; Proximity ligation assay (PLA); Pull-down assay; …402
41620240722_184716pk-gt-pharmacologymdPage 1229 of 1238[PK] > Parameters > Dose-PK-PD-Efficacy relationship in VivoBelow LOD data; [PK]; Parameters; Dose-PK-PD-Efficacy relationship in Vivo; …402
41720240722_184720pk-gt-pharmacologymdPage 1229 of 1239[PK] > Parameters > Dose-PK-PD-Efficacy relationship > Translational modeling (and prediction of clinical e…Translational Modeling Using A Reference Drug (Beloranib); ⊞ Translational modeling (and prediction of clinical efficacy) using a reference drug (that has clinical data); Questions; Beloranib PK an…; …402
41820240722_184723biomarkers-outcomesmdPage 1234 of 1242BIOMARKER > Validity > Milestones > Milestone: MBM OnlyBiomarker Validity, Milestones, BMx Milestone Matrix; [BIOMARKER]; Validity; Types of biomarkers; …402
41920240722_184726biomarkers-outcomesmdPage 1237 of 1244Validation > Biomarker milestones > BMx Assay Qualification > Biomarker type-specific definition of milestonesBiomarker Validation Milestones; to BAF; to BAQ; to BCS; …500
42020240722_184730biomarkers-outcomesmdPage 1237 of 1248Navigation pane shows: polygenic risk score > Steps of PRS STUDY; visible document/status context shows bio…Biomarker Development, Validation, and Proteomics Approach; Top Notes; Stages of Biomarker Development; Figure 17.1 Biomarker Development Pipeline; …600
42120240722_184733biomarkers-outcomesmdPage 1243 of 1252[BIOMARKER] > Validation > Analytical (=Technical) validationAnalytical (=Technical) validation; Biomarker development process; Analytical (=Technical) validation; Table Structure; …500
42220240722_184736biomarkers-outcomesmdPage 1243 of 1253[BIOMARKER] > Validation > Analytical (=Technical) validationAnalytical validation / assay validation notes; Analytical Validation Table Continuation; ELISA Validation; MJFF Phases of Assay Development; …600
42320240722_184740biomarkers-outcomesmdPage 1249 of 1258[BIOMARKER] > Examples of biomarker > Keytruda with MSI-H or dMMRExamples of biomarker — Keytruda PD-L1 / MSI-H / dMMR; Non-Small-Cell Lung Cancer (NCT01295827) — PD-L1 BM Define / Validation; Keytruda with MSI-H or dMMR; Indication; …402
42420240722_184743inflammationmdPage 1256 of 1262[GT] > AAV > Capsid (serotype)Keytruda / LoD-LLOQ / [GT] AAV / Capsid (serotype); [GT]; Scheme of a CMV virus; AAV; …400
42520240722_184746pk-gt-pharmacologymdPage 1260 of 1270[GT] > AAV > Clinical trial / Dose Selection (visible page spans multiple headings)AAV cassette structure, clinical trial, dose selection, and glossary; AAV / capsid table continuation; Cassette structure; Gene Therapy Approach for GBA-PD; …500
42620240722_184749pk-gt-pharmacologymdPage 1260 of 1271Life-cycle > Localization > Manufacturing > PK/PD strategyImmunosupressnats; Life-cycle; Localization; Manufacturing400
42720240722_184753pk-gt-pharmacologymdPage 1264 of 1277PK/PD strategy > Dose Scaling for GT > PromoterGT PK Comparison, Dose Scaling, And Promoter Section; GT PK comparison (continuation); Dose Scaling for GT; Species difference (Activity factor); …402
42820240722_184756molecular-biologymdPage 1270 of 1279[MOLECULAR BIOLOGY] > Mutations > Genotyping MethodsRe-dosing?; Regulatory Element; Route of Administration; Seroepidemiology (Seropositivity); …502
42920240722_184759pk-gt-pharmacologymdPage 1273 of 1282Study population > Type of gene therapy > Units (dose and transgene expression) > [PHARMACOLOGY]Below from Parkin GT LGE slidedeck; Study population; Type of gene therapy; Units (dose and transgene expression); …402
43020240722_184802pk-gt-pharmacologymdPage 1273 of 1284Units (dose and transgene…) > [PHARMACOLOGY] > Binding Affinity > PK-PD relationshipBinding Affinity; PD relationship400
43120240722_184806pk-gt-pharmacologymdPage 1276 of 1287[PHARMACOLOGY] > Binding Affinity > PK-PD relationship[PET]; Antibody radiolabelling; Parameters of PET ligand502
43220240722_184809pet-imagingmdPage 1279 of 1292[MOLECULAR BIOLOGY] > [PET] > Parameters of PET ligandBmax assessment in rats; Summary of Bmax and Kd value; Kd (흔히 Ki로 대치됨); Bmax/Kd binding potential; …602
43320240722_184813pet-imagingmdPage 1283 of 1295PK-PD relationship > [PET] > Antibody radiolabelling > Parameters of PET ligandVariability; Quantify target expression (Bmax) across species along with regional distribution to inform PET tracer viability; PET Tracer Viability:; Regional Tissue Distribution:; …502
43420240722_184816pet-imagingmdPage 1286 of 1298Parameters of PET ligand > Dose of PET tracer > PET Steering Committee > WorkflowAssay flow and criteria in lead optimization; Dose of PET tracer; PET Steering Committee; PET Ligand Development Steering Committee (PETSC); …300
43520240722_184819pet-imagingmdPage 1290 of 1301[PHARMACOLOGY] > Parameters of PET ligand > Dose of PET tracer > PET Steering Committee > WorkflowTarget validation workflow; Assay flow for 1st set of PET tracer candidates; Tentative flow for GCSi — Flow of PET tracer development (preclinical); Work Flow for PET Tracer Discovery; …402
43620240722_184823pet-imagingmdPage 1294 of 1309[MOLECULAR BIOLOGY] > [PET] > Parameters of PET ligandBiomarker Types and Stage Gates — Alignment with Asset Milestones; Biomarker type-specific definition of milestones; Comparison: in vitro vs in vivo; Preclinical study; …502
43720240722_184826pet-imagingmdPage 1300 of 1310Dose of PET tracer > PET Steering Committee > Workflow > Preclinical studySelectivity; PET probe discovery for HDAC6 (Cont’d); Which was better to evaluate specific binding: Kp.hippocampus or Hippocampus concentration; Non-imaging methods to triage selected compounds and…; …502
43820240722_184829pet-imagingmdPage 1300 of 1311PET Steering Committee > Workflow > Preclinical study > Clinical studyPreclinical study; In vivo PET imaging to assess regional distribution and blocking by drug candidate; In vivo PET imaging to assess regional distribution and blocking by drug candidate; Clinical s…400
43920240722_184833pk-gt-pharmacologymdPage 1304 of 1314Workflow > Budget/ResponsibilitiesHDAC6 PET Protocol End, Budget/Responsibilities, And Start Of Project Team Function Table; HDAC6 [18F]EKZ-001 Clinical PET Protocol (continuation); Budget/Responsibilities; Project Team — Function…302
44020240722_184836pet-imagingmdPage 1307 of 1318[MOLECULAR BIOLOGY] > [PET] > Regulatory Development Path PET ligandRegulatory Development Path PET ligand; Microdosing study; Outcome of PET radioligand study; Micro PET ligands; …401
44120240722_184840operationsmdPage 1307 of 1320Timeline > Wipe assay > [Budget] > Definition Budget categoryWipe assay; [Budget]; Definition Budget category300
44220240722_184843operationsmdPage 1313 of 1324[Budget] > Definition Budget category > Cost center > FY19Transfer of Budget; Cost center; FY19; NS Translation – Cross-Functional FY 19 Budget – OCT 2019 Update500
44320240722_184847operationsmdPage 1313 of 1331FY21 > FY21 MRPFY21 NSTM-Shonan Budget Detail And FY21 MRP Start; FY20; NSTM-Shonan budget; FY19 actual spend vs. the amended FY20 plan (after Q1-Q2 program-specific budget allocation), as of August 2020; …402
44420240722_184850operationsmdPage 1320 of 1332[Budget] > FY21 > FY21 B. KPI-linked proje…A FY21 draft allocation; B. KPI-linked projects and tentative budget; C FY21 draft allocation by Unit/Labs; NSTM:; …301
44520240722_184854molecular-biologymdPage 1324 of 1333[Glossary] > Amino Acid CodesAmino Acid Codes202
44620240722_184857molecular-biologymdPage 1330 of 1333FY21 B. KPI-linked proje… > FY21 MRP > Glossary > Amino Acid Codes400
44720240722_184901molecular-biologymdPage 1333 of 1333FY21 B. KPI-linked projects > FY21 MRP > Glossary > Amino Acid CodesLogarithm301

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