The role
0401Define and execute integrated translational strategies across neurodegeneration programs
02Lead translational components from preclinical → Phase 1/2 clinical studies
03Drive program progression through key decision gates (e.g., IND, TDP, IDPA)
04Ensure strong mechanistic linkage between target biology, biomarkers, and clinical endpoints
05Collaborate with Biomarker Development colleagues toward development and validation of: Molecular biomarkers (tissue, blood, CSF where applicable)
06Functional endpoints (strength, mobility, disease-specific measures)
07Patient-reported outcomes (PROs)
08Establishing quantitative biomarkers to support: Target engagement
09Disease modification
10Dose selection and PoC decisions
11Integrate biomarker strategy into clinical protocols and operational plans
12Drive early biomarker de‑risking , a key priority in neuromuscular programs
13Co‑lead design and execution of early clinical studies
14Define: Dose escalation strategy
15Exposure–response framework
16Translational endpoints (biopsy, imaging, functional)
17Ensure TM leadership within Clinical Trial Teams (CTTs) for: Trial design
18Execution oversight
19Data interpretation and decision-making
20Serve as core Translational Medicine representative within global project teams
21Partner closely with: Clinical Development
22Biomarker Development
23Clinical Pharmacology (PKS)
24Preclinical Safety (PCS)
25Regulatory
