AD/ Principal Scientist CMC AAV Gene Therapy
About The Position
Ocugen is seeking an experienced CMC leader to drive BLA-stage readiness for AAV gene therapy programs. The role serves as the strategic technical interface between laboratory development, GMP manufacturing, regulatory affairs, and program leadership, supporting the transition from clinical development to licensure. It leads CMC integration across AAV drug substance production, drug product formulation and fill-finish, analytical characterization, process validation, comparability, and stability programs. A core responsibility is authoring and coordinating Module 3 content for a BLA and supporting regulatory interactions through approval. The position requires expertise in AAV manufacturing science, regulatory expectations, and lifecycle management. The position is based in person in an office setting at the Main Corporate Office in Malvern, Pennsylvania. The incumbent may additionally need to be available outside regular hours for priority business needs.
Requirements
- Ph.D. (preferred) or Master’s degree in Biochemical Engineering, Biotechnology, Pharmaceutical Sciences, or a related field.
- 10+ years of experience in biologics or AAV gene therapy CMC development.
- Direct experience leading or significantly contributing to BLA-stage submissions.
- Deep knowledge of AAV manufacturing platforms.
- Knowledge of potency assay development and validation.
- Knowledge of PPQ and process validation for biologics.
- Knowledge of stability and shelf-life justification.
- Knowledge of regulatory expectations for gene therapy products.
- Experience supporting FDA meetings and pre-approval inspections.
- Strong program leadership and cross-functional execution skills.
Nice To Haves
- Experience with at least one AAV program through BLA submission or approval.
- Expertise in empty/full capsid analytics and advanced characterization.
- Experience with multi-site technology transfer or commercial launch preparation.
- Familiarity with global filings, including EMA and PMDA.
Responsibilities
- Lead development and authoring of CMC sections (Module 3) for BLA submissions.
- Integrate drug substance, drug product, analytical, validation, stability, and control-strategy information into cohesive regulatory narratives.
- Develop and justify control strategies and specification lifecycle approaches, comparability frameworks, and process validation and PPQ strategies.
- Coordinate cross-functional data readiness to meet BLA timelines.
- Lead preparation of responses to FDA information requests and late-cycle review questions, and provide potential Advisory Committee support.
- Oversee phase-appropriate transition to commercial-scale AAV manufacturing.
- Provide strategic oversight of process characterization studies, viral clearance where applicable, PPQ protocol development and execution, and Continued Process Verification planning.
- Ensure the control strategy is statistically justified and aligned with ICH Q8/Q9/Q10 principles.
- Drive readiness for pre-approval inspection (PAI).
- Provide technical leadership for AAV upstream processes, downstream purification, 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).
- Oversee formulation optimization, aseptic fill-finish readiness, and container closure integrity.
- Ensure the stability program supports shelf-life justification at BLA.
- Drive extractables/leachables, shipping validation, and in-use stability readiness.
- Lead comparability strategy across development phases, manufacturing scale changes, site transfers, and CDMO transitions.
- Develop risk-based justification where formal comparability protocols were not historically implemented and ensure analytical depth supports regulatory defensibility.
- Serve as primary CMC integration lead across Process Development, Analytical Development, MSAT, Quality, and Regulatory Affairs.
- Act as senior technical liaison with CDMOs, aligning on validation, documentation, and regulatory positioning.
- Maintain disciplined communication pathways for regulatory-sensitive decisions.
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What This Job Offers
Job Type
Full-time
Career Level
Senior
