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 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 into and through the clinic. This individual will be part of a fast-paced cross-functional team to bring gene therapies through development.
Accountabilities and Responsibilities
• 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, publication strategy.
• Ensure adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.
-PhD or PharmD required
-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 start-up and scaling environment.
-Understanding of submission of INDs and marketing approval-directed filing(s) (BLAs, NDAs, and MAAs).
•-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 deliver professional and quality work products, while working on multiple projects.