At Neurogene, we are working to provide 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 recruiting for a Vice President of Clinical Development to lead up to 2 parallel development programs in neurologic genetic disorders that will enter the clinic in the near term. Reporting to the Senior Vice President of Clinical Development, the Vice President of Clinical Development will be an integral member of the Neurogene leadership team, contributing significantly to the advancement of programs into and through the clinic, and be a part of a fast-paced, cross-functional team to bring these therapies through development. The direct responsibilities of this position will cover Phase 1 through Phase 3 and post-approval activities, expanding and advancing the Company’s programs from early clinical trial development to proof-of-concept and commercialization. This Vice President will be in a high profile and high impact position, responsible for executing and delivering on critical clinical milestones, and will also be integral to the clinical development strategy. The individual hired for this position will, therefore, have a significant influence shaping the future of the Company.
Accountabilities and Responsibilities
- Leading clinical development programs from the late preclinical stage, to registration and completion of post-approval commitments.
- Working cross-functionally with Clinical Operations and/or vendors to successfully execute clinical trials and natural history studies. These activities may consist of coordination, collection, and analysis of clinical data and reporting, and providing ongoing medical monitoring for clinical trials, including assessment of eligibility criteria, toxicity management, and drug safety surveillance.
- 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.
- Interacting with global regulatory agencies and having responsibility for authoring and/or reviewing relevant Pre-IND, IND, and BLA sections and generating responses.
- Close collaboration 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.
- Must be able to critically evaluate and understand the unmet medical needs, outcome measures, and treatment options in various rare neurologic disorders.
- 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.
- Representing Neurogene externally where necessary through publications, presentations at scientific meetings, and congresses, both domestically and internationally.
- Working collaboratively to assist with business development and pipeline activities.
- Ensuring adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.
- Education • MD is required
- Successful track record of designing and leading clinical studies, preferably in rare diseases.
- Translational medicine experience a plus.
- Able to run multiple clinical programs independently and work collaboratively with internal/external key stakeholders to ensure high-quality clinical protocols.
- Lead study teams to support what is expected to be a streamlined development process.
- Experience in pediatrics, neurology, rare diseases, and/or gene therapy ideal.
- 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.
- Minimum of 10 years of substantive experience in the pharmaceutical/biotechnology industry, and an in-depth understanding of pharmaceutical regulatory requirements and their impact on the development of clinical trials and BLAs.
- MD with biopharmaceutical industry experience and translational medicine experience.
- A proven success record in gene therapy and/or rare diseases is preferred.
- Successful submission of INDs and submission of marketing approval-directed filing(s) (BLAs, NDAs, and MAAs) is preferred.
- Ability to create an active network with KOLs, key research centers, patient advocacy groups, and a variety of patient care organizations.
- Experience in designing and managing clinical trials and clinical development strategy.
- Proven leadership and critical decision-making skills.
- Exceptional analytical, critical thinking, and problem-solving abilities; strong attention to detail.
- Demonstrated organizational and collaboration skills.
- Ability to deliver professional and quality work products while working on multiple projects.
- Desire and ability to work in a fast-paced, dynamic start-up and scaling environment.
- Thorough knowledge of clinical research concepts, practices, and GCP and ICH Guidelines.