Executive Director, Clinical Development

GEMMABio is advancing a diverse portfolio of gene therapy programs across multiple therapeutic areas, including neuromuscular, cardiovascular, oncology, metabolic, and infectious diseases.

Summary

Reporting to the Chief Medical Officer, (CMO) The Executive Director, Clinical Development, will provide tactical oversight for the clinical/medical aspects of all phases of the clinical drug development process. The Executive Director will be involved in the design, development and execution of Clinical Development Plans (CDPs), clinical trials and protocols in accordance with Good Clinical Practice (GCP) regulations. The person will lead and contribute to the analysis of study results, the assessment of the extent to which the studies have achieved clinical, regulatory and commercial objectives and the documentation of study results.

As needed, the candidate may be assigned as the primary clinical/medical representative in meetings with external stakeholders, partners or other external audiences as needed. This includes, but may not be limited to, Key Opinion Leaders (KOLs), investigators, regulatory authorities, partners, vendors, consultants, etc. As needed, may author or review clinical/medical-related materials intended for external audiences.

• Contributes to, reviews and approves study protocols, protocol amendments, study manuals, Investigator Brochures (IBs), Clinical Study Reports (CSRs), Informed Consent Forms (ICFs), Case Report Forms (CRFs) and other clinical documents as required for the conduct of clinical trials.
• Participates in KOL and investigator sites selection for inclusion in study/studies.
• Prepare weekly and/or monthly study update reports which contain detailed information on enrollment data and adverse events as well as other general information relating to the progress of the study.
• Interprets, summarizes and documents clinical data for CSRs, regulatory documents (e.g. INDs, Annual Reports, NDAs, MAAs, etc.) manuscripts, promotional material and other publications as required.
• Serve as an early stage clinical development lead and expected to partner with Translational Research unit to ensure clinical input is integrated in nonclinical and NHP studies to support TPP.

Key Elements

• Clinical Strategy – In partnership with Chief Medical Officer (CMO), support the clinical development strategy for programs targeting rare and ultra rare neurodegenerative genetic disorders
• Trial Design and Execution – Lead activities related to Phase 1-4 clinical trials to deliver on the strategic vision and address patient needs (e.g., authoring and reviewing protocols, investigator brochures, and clinical study reports; CRO management).
• Medical Monitoring oversight– Define, plan, and provide dedicated medical monitoring support, including the evaluation of participant eligibility, safety, and efficacy across ongoing and completed studies in Neuromuscular, CNS, Metabolic & other rare disease designated focus areas.
• Pharmacovigilance & Data Analysis – Review adverse events, monitor real-time safety data, prepare analyses and clinical input for regulatory submissions, and lead data interpretation in partnership with pharmacovigilance CRO partners
• DMC & Safety Governance – May serve as a clinical representative for Data Monitoring Committee (DMC) safety reviews, evaluating potential dose-limiting toxicities and ensuring CRO pharmacovigilance oversight across the clinical portfolio.

Qualifications