Principal Clinical Scientist

Principal Clinical Scientist

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

Neurogene is seeking a Principal Clinical Scientist to support the Clinical Development Team in collaborating with program partners across Development, Operations, Regulatory and Pre-Clinical, contributing significantly to protocol development, study execution, review and interpretation of scientific and clinical data. This role will perform ongoing review and analysis of study data to support the advancement of programs focused on neurologic genetic disorders. This individual will be part of a fast-paced cross-functional team to bring gene therapies through development.

Requirements

• Review and analysis of study data.
• Work cross-functionally to support the successful execution of clinical trials and natural history studies.
• Provide support for scientific issues that may arise during study execution.
• Identify, critically review and summarize literature that addresses specific topics related to scientific, medical, drug development, neurological disorders, gene therapies and rare disease.
• Prepare and/or review of data listings, summary tables, study results, study reports, and clinical/regulatory/safety documents, investigator brochures, and clinical development plans.
• Review relevant Pre-IND, IND, and BLA sections for the Development Team.
• Collaborate closely with academic institutions associated with the clinical development programs.
• Critically evaluate and understand the unmet medical needs in various rare neurologic disorders, outcome measures and treatment options.
• Design and support clinical trials in collaboration with Clinical Operations.
• Partner with internal 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.
• Collaborate with Medical Affairs in developing medical slide decks, educational materials, and publication strategy.
• Ensure adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.

Minimum Requirements

Education

• PhD or PharmD required

Experience

• Minimum of 5 years of experience in the pharmaceutical/biotechnology industry, and an in-depth understanding of scientific and clinical data, clinical study designs and clinical trial outcomes.
• Familiarity with and exposure to the implementation of rare diseases natural history studies and gene therapy trials.
• Authoring of key documents in clinical programs: clinical study reports, clinical trial documents.
• 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 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 a 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.
• 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.