Brief Description
Company Background
The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. This transformative work is driven by our highly collaborative people who come together each day to bring us closer to achieving our mission. As an employee of Neurogene, you will be part of building a culture that honors patient and caregiver mindsets and nurtures innovation, creative problem solving and a strong sense of purpose.
Position Overview
The Medical Director, Clinical Development for NGN-401, at Neurogene will serve as the medical monitor in support of NGN-401 for Rett syndrome. This individual will support the execution of the clinical study for NGN-401 for Rett syndrome and will be directly accountable to the Clinical Development Lead for NGN-401 and the CMO.
Requirements
Collaborate closely with cross-functional teams. including Clinical Operations, Regulatory Affairs, Pharmacovigilance, Biostatistics, Manufacturing, and with vendors to successfully execute clinical trials.
Activities may consist of coordination, collection and analysis of clinical data and reporting; serving as the medical monitor, and assessment of eligibility criteria, safety management, and drug safety surveillance, among other activities.
Support the Clinical Development Lead and CMO in designing and managing the trial in collaboration with Clinical Operations. Author protocols and relevant sections in regulatory documents.
Support the preparation and submission of Clinical sections of regulatory filings, including Investigational New Drug (IND) applications, clinical trial applications (CTA), working closely with regulatory agencies (FDA, EMA, etc.) to ensure adherence to guidelines and timelines.
Serve as a scientific and clinical expert for the NGN-401 program, ensuring clinical and preclinical data are interpreted correctly to inform ongoing program decisions.
Actively participate in meetings with external stakeholders, including KOLs, regulatory bodies, and CROs.
Build and maintain relationships with experts to improve clinical trial design and execution, fostering constructive scientific discussions on study design, conduct, and results interpretation.
Engage with thought leaders, investigators, cooperative groups, and other experts in constructive scientific and clinical dialogue around study design, study conduct, and interpretation of clinical results.
Collaborate closely with academic institutions associated with the clinical development program. Manage 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.
Collaborate with Medical Affairs in developing key messaging, medical slide decks, educational materials, and publication strategy.
Partner 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.
Ensure adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.
Minimum Requirements
Education
MD or DO required; translational experience a plus
Experience
Minimum of 6 years of substantive experience in the pharmaceutical/biotechnology industry, and an in-depth understanding of clinical study designs, successful execution of clinical studies, experience in medical monitoring, drafting protocols, authoring clinical sections in Regulatory documents.
Experience in preclinical to clinic translation is desired
Contributing to the design and implementation of rare disease gene therapy trials.
Authoring key documents in clinical programs: clinical study reports, clinical trial documents, for example.
Experience in gene therapy is required; experience in pediatrics, neurology, immunology, and/or rare diseases is a plus.
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).
Continuous strong focus on meeting or exceeding expectations in executing, deliverables, performance and results.
Ability to track and measure performance against defined metrics.
Self-starter with high degree of energy, independence, initiative, and self-motivation, as illustrated by the ability to instill a sense of pace and urgency into the team.
Someone who moves fast and decisively in a balanced manner, with a passion for the growing company.
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 leverage an active network of KOLs, key research centers, patient advocacy groups and a variety of 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 work collaboratively in a highly matrixed environment.