Neurogene Inc.
Company Background
At Neurogene, we focus on bringing life‑changing genetic medicines to patients and families affected by rare, devastating neurological diseases. Our clinical‑stage company is developing NGN‑401 for the treatment of Rett syndrome. We use our proprietary EXACT transgene regulation technology and internal expertise to develop novel approaches and treatments that address the limitations of conventional gene therapy in central nervous system disorders. Our wholly owned and fully‑operational GMP‑capable manufacturing facility supports vector production from research through clinical grade and is designed for commercial‑grade production. Neurogene is also traded on the NASDAQ Global Market.
Position Overview We are seeking a Director, CMC Regulatory Affairs to join the Regulatory Affairs CMC team and support a novel rare disease CNS pipeline. The individual will provide regulatory strategy and support to the CMC/Manufacturing Teams at our gene therapy manufacturing facility in Houston, developing, planning, and executing the regulatory strategy to facilitate the company’s short and long‑term objectives.
Accountabilities and Responsibilities
Provide CMC regulatory input to cross‑functional teams, including Technical Operations, Process Development, Quality, and Clinical Development.
Contribute to global regulatory development plans and participate in CMC strategy discussions at project governance meetings.
Partner with Process Development, Analytical Development, Quality, and Manufacturing teams to align regulatory strategies with technical and operational objectives in compliance with all applicable regulations and guidance.
Anticipate and proactively identify regulatory risks, supporting mitigation strategies and recommendations.
Support cross‑functional problem‑solving and contribute to decision‑making on complex regulatory and CMC development challenges.
Ensure compliance with applicable laws, regulations, and guidelines by monitoring and interpreting the regulatory landscape, evolving guidance, and external trends relevant to gene therapy.
Lead preparation, review, and submission of CMC components of INDs, CTAs, BLAs, MAAs, and related amendments/supplements for gene therapy products in accordance with global regulatory requirements.
Support responses to health authority questions, preparation of briefing packages, and interactions with regulators on CMC matters, including preparation of internal teams for these engagements.
Collaborate with clinical regulatory colleagues to ensure cohesive and compliant submissions across all modules.
Support the development and maintenance of Regulatory Affairs programs, policies, and procedures.
Minimum Requirements
Bachelor’s degree in life sciences or a related field required.
Advanced degree (Ph.D., M.S., or equivalent) in Molecular Biology, Biochemistry, Pharmaceutical Sciences, or a related discipline preferred.
Experience
10+ years of experience in Regulatory Affairs CMC with at least 5 years focused on gene therapy, viral vectors, or biologics.
Proven success in developing and executing global regulatory CMC strategies for advanced therapy medicinal products (ATMPs) or biologics.
Strong track record of leading CMC submissions (IND/CTA through BLA/MAA) for gene therapy or biologics.
In‑depth understanding of biologics manufacturing, characterization, control strategies, and analytical development.
Experience interacting directly with global health authorities.
Prior leadership experience managing teams or cross‑functional project workstreams.
Knowledge and Skills
Expert knowledge of global CMC and gene therapy‑specific regulations and guidance (FDA CBER, EMA ATMP, ICH Q‑series).
Strong scientific understanding of viral vectors (AAV, lentivirus, etc.), manufacturing technologies, and quality control principles.
Extensive experience with applicable regulatory requirements, including GxPs, and previous interactions with FDA, EMA, and other regulatory agencies.
Exceptional interpersonal and communication skills, with the ability to interact effectively with senior leaders, cross‑functional teams, and colleagues at all levels while exercising sound regulatory judgment in complex situations.
Strong project management, organizational, and leadership abilities, with the capacity to prioritize and drive initiatives efficiently in a fast‑paced environment.
Self‑starter with high energy, initiative, and motivation, able to instill a sense of urgency and pace within the team.
Demonstrated ability to work decisively and collaboratively in a rapidly evolving, high‑growth environment.
Passionate, creative, and collaborative, with a willingness to take prudent risks and contribute to a mission‑driven company focused on transformative treatments for patients.
Excellent written and verbal communication skills.
Travel
Travel to Houston will be required.
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Position Overview We are seeking a Director, CMC Regulatory Affairs to join the Regulatory Affairs CMC team and support a novel rare disease CNS pipeline. The individual will provide regulatory strategy and support to the CMC/Manufacturing Teams at our gene therapy manufacturing facility in Houston, developing, planning, and executing the regulatory strategy to facilitate the company’s short and long‑term objectives.
Accountabilities and Responsibilities
Provide CMC regulatory input to cross‑functional teams, including Technical Operations, Process Development, Quality, and Clinical Development.
Contribute to global regulatory development plans and participate in CMC strategy discussions at project governance meetings.
Partner with Process Development, Analytical Development, Quality, and Manufacturing teams to align regulatory strategies with technical and operational objectives in compliance with all applicable regulations and guidance.
Anticipate and proactively identify regulatory risks, supporting mitigation strategies and recommendations.
Support cross‑functional problem‑solving and contribute to decision‑making on complex regulatory and CMC development challenges.
Ensure compliance with applicable laws, regulations, and guidelines by monitoring and interpreting the regulatory landscape, evolving guidance, and external trends relevant to gene therapy.
Lead preparation, review, and submission of CMC components of INDs, CTAs, BLAs, MAAs, and related amendments/supplements for gene therapy products in accordance with global regulatory requirements.
Support responses to health authority questions, preparation of briefing packages, and interactions with regulators on CMC matters, including preparation of internal teams for these engagements.
Collaborate with clinical regulatory colleagues to ensure cohesive and compliant submissions across all modules.
Support the development and maintenance of Regulatory Affairs programs, policies, and procedures.
Minimum Requirements
Bachelor’s degree in life sciences or a related field required.
Advanced degree (Ph.D., M.S., or equivalent) in Molecular Biology, Biochemistry, Pharmaceutical Sciences, or a related discipline preferred.
Experience
10+ years of experience in Regulatory Affairs CMC with at least 5 years focused on gene therapy, viral vectors, or biologics.
Proven success in developing and executing global regulatory CMC strategies for advanced therapy medicinal products (ATMPs) or biologics.
Strong track record of leading CMC submissions (IND/CTA through BLA/MAA) for gene therapy or biologics.
In‑depth understanding of biologics manufacturing, characterization, control strategies, and analytical development.
Experience interacting directly with global health authorities.
Prior leadership experience managing teams or cross‑functional project workstreams.
Knowledge and Skills
Expert knowledge of global CMC and gene therapy‑specific regulations and guidance (FDA CBER, EMA ATMP, ICH Q‑series).
Strong scientific understanding of viral vectors (AAV, lentivirus, etc.), manufacturing technologies, and quality control principles.
Extensive experience with applicable regulatory requirements, including GxPs, and previous interactions with FDA, EMA, and other regulatory agencies.
Exceptional interpersonal and communication skills, with the ability to interact effectively with senior leaders, cross‑functional teams, and colleagues at all levels while exercising sound regulatory judgment in complex situations.
Strong project management, organizational, and leadership abilities, with the capacity to prioritize and drive initiatives efficiently in a fast‑paced environment.
Self‑starter with high energy, initiative, and motivation, able to instill a sense of urgency and pace within the team.
Demonstrated ability to work decisively and collaboratively in a rapidly evolving, high‑growth environment.
Passionate, creative, and collaborative, with a willingness to take prudent risks and contribute to a mission‑driven company focused on transformative treatments for patients.
Excellent written and verbal communication skills.
Travel
Travel to Houston will be required.
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