AD/ Principal Scientist CMC AAV Gene Therapy

OCUGEN OPCO INC; Malvern (Chester County); On-site; USD 180,000 - 280,000; Full time

Job summary

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

You will lead CMC integration across DS, DP, analytical characterization, validation, and stability programs, coordinating data readiness to meet BLA timelines and preparing responses to FDA

Qualifications

• 10+ years in biologics or AAV gene therapy CMC development.
• Experience leading or contributing to BLA submissions.
• Deep knowledge of AAV manufacturing platforms.
• Strong program leadership and cross-functional execution.

Responsibilities

• Lead development and authoring of Module 3 content for BLA submission.
• Oversee DS/DP, analytical, validation, stability programs integration.
• Drive pre-approval inspection readiness and regulatory responses.
• Ensure process validation and CPV align with ICH Q8/Q9/Q10.

Skills

CMC leadership

Regulatory submissions

Cross-functional leadership

Gene therapy

AAV manufacturing

Education

PhD in Biochemical Engineering

MSc in Biotechnology

Job description

Position SummaryWe 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.ResponsibilitiesBLA 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 lifecycleComparability frameworks (process changes, site transfers, scale-up)Process validation and PPQ strategyCoordinate 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 ReadinessOversee phase-appropriate transition to commercial-scale AAV manufacturing.Provide strategic oversight of:Process characterization studiesViral clearance (if applicable)PPQ protocol development and executionContinued Process Verification (CPV) planningEnsure control strategy is statistically justified and aligned with ICH Q8/Q9/Q10 principles.Drive readiness for pre-approval inspection (PAI).Drug Substance (AAV) OversightProvide technical leadership in:Upstream (cell expansion, transfection/infection systems)Downstream purification (chromatography, TFF, clarification)Empty/full capsid ratio controlVector genome integrity and potency assaysEnsure manufacturing consistency across clinical and commercial batches.Lead technical assessment of critical process parameters (CPPs) and critical quality attributes (CQAs).Drug Product & Stability StrategyOversee 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 ManagementLead comparability strategy across:Phase 1 to Phase 3 evolutionManufacturing scale changesSite transfers or CDMO transitionsDevelop risk-based justification when formal comparability protocols were not historically implemented.Ensure analytical depth supports regulatory defensibility.Cross-Functional & CDMO InterfaceServe as primary CMC integration lead across:Process DevelopmentAnalytical DevelopmentMSATQualityRegulatory AffairsAct as senior technical liaison with CDMOs, ensuring alignment on validation, documentation, and regulatory positioning.Maintain disciplined communication pathways for regulatory-sensitive decisions.Ph.D. (preferred) or Master’s in Biochemical Engineering, Biotechnology, Pharmaceutical Sciences, or related field.10+ years in biologics or AAV gene therapy CMC development.Direct experience leading or significantly contributing to BLA-stage submissions.Deep knowledge of:AAV manufacturing platformsPotency assay development and validationPPQ and process validation for biologicsStability and shelf-life justificationRegulatory expectations for gene therapy productsExperience supporting FDA meetings and pre-approval inspections.Strong program leadership and cross-functional execution skills.Preferred ExperienceExperience with at least one AAV program through BLA submission or approval.Expertise in empty/full capsid analytics and advanced characterization.Experience with multi-site tech transfer or commercial launch preparation.Familiarity with global filings (EMA, PMDA).Working ConditionsThis position operates in an office setting, in person. Job may additionally require incumbent to be available outside of these hours to handle priority business needs.Ocugen is an equal opportunity employer. In order to provide equal employment and advancement opportunities to all individuals, we make all of our employment decisions based upon merit, qualifications, abilities, and an individual’s conduct and performance. We will not make any of our decisions, and will not discriminate against any employee or applicant, on the basis of race, color, religion, creed, national origin or ancestry, ethnicity, sex (including pregnancy), gender (including sexual orientation, gender identity and status as a transgender or transsexual individual), age, physical or mental disability, citizenship, past, current or prospective service in the uniformed services, genetic information, or any other characteristic protected from discrimination under the law. Ocugen complies with applicable federal, state and local laws governing nondiscrimination in employment.