Position Summary

We are seeking an experienced CMC leader to drive BLA-stage readiness for AAV gene therapy programs. This role serves as the strategic technical interface between laboratory development, GMP manufacturing, regulatory affairs, and program leadership to ensure seamless transition from clinical development to licensure.

The individual will lead CMC integration across drug substance (AAV vector production), drug product (formulation and fill-finish), analytical characterization, process validation (PPQ), comparability strategy, and stability programs. A core responsibility of this role is authoring and coordinating high-quality Module 3 content for BLA submission and supporting regulatory interactions through approval.

This is a high-visibility role requiring deep expertise in AAV manufacturing science, regulatory expectations, and lifecycle management.

 

Responsibilities 

BLA Strategy & Authoring (Module 3 Lead)

• Lead development and authoring of CMC sections (Module 3) for BLA submission.


• Integrate DS, DP, analytical, validation, stability, and control strategy into cohesive regulatory narratives.


• Develop and justify:
  
  
  
  • Control strategy and specification lifecycle
  
  
  • Comparability frameworks (process changes, site transfers, scale-up)
  
  
  • Process validation and PPQ strategy
  
  
  
  


• Coordinate data readiness across functions to meet BLA timelines.


• Lead preparation of responses to FDA information requests, late-cycle review questions, and potential Advisory Committee support.

Process Validation & Commercial Readiness

• Oversee phase-appropriate transition to commercial-scale AAV manufacturing.


• Provide strategic oversight of: 
  
  
  
  • Process characterization studies
  
  
  • Viral clearance (if applicable)
  
  
  • PPQ protocol development and execution
  
  
  • Continued Process Verification (CPV) planning
  
  
  
  


• Ensure control strategy is statistically justified and aligned with ICH Q8/Q9/Q10 principles.


• Drive readiness for pre-approval inspection (PAI).


• 
  

  Drug Substance (AAV) Oversight

  
  


• Provide technical leadership in:


• Upstream (cell expansion, transfection/infection systems)


• Downstream purification (chromatography, TFF, clarification)


• Empty/full capsid ratio control


• Vector genome integrity and potency assays


• Ensure manufacturing consistency across clinical and commercial batches.


• Lead technical assessment of critical process parameters (CPPs) and critical quality attributes (CQAs).


• 
  

  Drug Product & Stability Strategy

  
  


• Oversee formulation optimization, aseptic fill-finish readiness, and container closure integrity.


• Ensure stability program supports shelf-life justification at BLA.


• Drive extractables/leachables, shipping validation, and in-use stability readiness.


• 
  

  Comparability & Lifecycle Management

  
  


• Lead comparability strategy across:


• Phase 1 to Phase 3 evolution


• Manufacturing scale changes


• Site transfers or CDMO transitions


• Develop risk-based justification when formal comparability protocols were not historically implemented.


• Ensure analytical depth supports regulatory defensibility.


• Cross-Functional & CDMO Interface


• Serve as primary CMC integration lead across:
  
  
  
  • Process Development
  
  
  • Analytical Development
  
  
  • MSAT
  
  
  • Quality
  
  
  • Regulatory Affairs
  
  
  
  


• Act as senior technical liaison with CDMOs, ensuring alignment on validation, documentation, and regulatory positioning.


• Maintain disciplined communication pathways for regulatory-sensitive decisions.