At Neurogene, we are working to develop transformative medicines to improve the lives of neurologically impaired children and their families. We envision a world in which all families have access to genetic medicines, even if the disorder is exceedingly rare. For children, our goal is to enable them to have a healthier future. For families, we want to make their lives better by easing the challenges of caring for someone with a devastating neurological disorder. As an employee of Neurogene, you will be part of a culture helping to make this dream a reality, collaborating with people in a start-up environment whose mission is to improve the lives of the patients we serve and make the world a better place.
Neurogene is seeking a Director, Clinical Development, to support the Development Leadership Team, contributing significantly to the advancement of programs focused on neurologic genetic disorders into and through the clinic. This individual will be part of a fast-paced cross-functional team to bring therapies through development. The direct responsibilities of this position will cover Phase 1 through Phase 3 and post-approval activities, expanding and advancing programs from early clinical trial development to proof-of-concept and commercialization. This role will be responsible for executing and delivering on critical clinical milestones.
Accountabilities and Responsibilities
- Executing clinical development programs from late preclinical stage to registration and completion of post-approval commitments.
- Working cross-functionally with Clinical Operations and vendors to successfully execute clinical trials and natural history studies. Activities may consist of coordination, collection, and analysis of clinical data and reporting, providing ongoing medical monitoring for clinical trials, including assessment of eligibility criteria, toxicity management, and drug safety surveillance, among other activities.
- Managing the preparation and/or review of data listings, summary tables, study results, study reports, and clinical/regulatory/safety documents, investigator brochures, and clinical development plans.
- Reviewing relevant Pre-IND, IND, and BLA sections and generating responses for the Development Leadership Team.
- Collaborating closely with academic institutions associated with the clinical development program.
- Engaging with thought leaders, investigators, cooperative groups, and other experts in constructive scientific and clinical dialog around study design, study conduct, and interpretation of clinical results.
- Critically evaluating and understanding the unmet medical needs in various rare neurologic disorders, outcome measures, and treatment options.
- Designing and managing clinical trials in collaboration with Clinical Operations.
- Authoring protocols and relevant sections in regulatory documents.
- Reviewing patient-facing materials for accuracy
- Partnering with internal and external key stakeholders to develop manuscripts for publication in peer-reviewed journals and preparation of presentations for scientific conferences as well as for clinical study investigator meetings and expert clinical advisory meetings.
- Collaborating with Medical Affairs in developing medical slide decks, educational materials, publication strategy.
- Ensuring adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.
- MD and/or Ph.D. required; translational experience is a plus.
- Minimum of 5 years of substantive experience in the pharmaceutical/biotechnology industry, and an in-depth understanding of clinical study designs, managing clinical studies, drafting protocols, authoring clinical sections in Regulatory documents.
- Contributing to the design and implementation of rare diseases, natural history studies, and gene therapy trials.
- Authoring key documents in clinical programs: clinical study reports, clinical trial documents, for example.
- Ability to work collaboratively and establish ongoing relationships with internal cross-functional team members and external key stakeholders to ensure successful program advancement. Experience in gene therapy and/or rare diseases is preferred.
- Experience in pediatrics, neurology, rare diseases, and/or gene therapy would be ideal.
Skills, Knowledge & Attributes
- Thorough knowledge of clinical research concepts, practices, and GCP and ICH Guidelines.
- Desire and ability to work in a fast-paced, dynamic start-up and scaling environment.
- Understanding of submission of INDs and marketing approval-directed filing(s) (BLAs, NDAs, and MAAs).
- Experience designing and managing clinical trials.
- Passionate, collaborative, creative, willing to take prudent risks, and motivated by the creation of a company focused on transformative treatments for patients and their families.
- Ability to engage a network of KOLs, key research centers, patient advocacy groups, and various patient care organizations.
- Critical and strategic decision-making skills.
- Exceptional analytical, critical thinking, and problem-solving abilities.
- Strong attention to detail.
- Demonstrated organizational and collaboration skills.
- Able to write and present clearly to a diverse audience, including clinicians, caregivers, regulatory agencies, among others.
- Ability to deliver professional and quality work products while working on multiple projects.