Medical Director, Genetic Medicines

Purpose

Clinical Research Physician (CRP), Gene Therapy Medical Development—supports clinical development of gene therapies for sensory and rare disorders, including study design, execution, and interpretation.

Core Responsibilities

• Clinical planning: Collaborate with Discovery; align global clinical strategy/plans; stay current on preclinical/clinical data.
• Trial execution/support: Plan/review clinical documents (protocols, consent forms, final reports, submissions); provide input/oversight to clinical development plans; collaborate on local trials (sample size, timelines, grants, governance); review/approve risk profiles; support investigator selection; ensure study initiation administrative requirements are completed (REB, informed consent, regulatory approvals) per GCP/local laws; support study start-up/training; serve as resource to operations/monitors/investigators/REB; monitor patient safety and track/follow adverse events.
• Scientific data dissemination/exchange: Ensure compliance with data dissemination policies; address external HCP scientific needs; support trial registry reporting; plan symposia/advisory boards; support medical letters and materials; review/approve slide sets and publications; build collaborations with external experts/thought leaders; support congress activities and data dissemination.
• Regulatory support: Participate in label change reviews; provide medical expertise to regulators; support regulatory reports (e.g., BLAs/annual reports, advisory committees, registration) and resolve regulatory issues; participate in advisory committees; support risk management planning.
• Customer/business support (pre/post launch): Support PRA organization with clinical input; develop medical strategies for commercialization; engage with payers/patients/HCPs; contribute to promotional material review and patient journey definition.
• Scientific/technical: Critically evaluate literature/competitive data; ensure team scientific training; act as protocol expert; attend symposia; pursue extramural scientific experiences.

Minimum Qualifications

• MD or DO; board eligible/certified in relevant field (or comparable post-medical school clinical training for country of hire).

Additional Preferences (Desired)

• Experience in gene therapy and/or rare disease Medical Affairs or Patient Advocacy.
• 3–5+ years clinical research in pharma or academic setting; knowledge of drug development, GCP, regulatory requirements; global trials experience.
• Strong scientific acumen; ability to communicate complex science; advanced presentation/computer skills; leadership; self-directed multi-tasking; strong communication/interpersonal/teamwork/negotiation; ability to influence across functions; domestic/international travel; fluent English.

Benefits

• Full-time employees eligible for company bonus; comprehensive benefits including 401(k) and pension; medical/dental/vision/prescription; flexible benefits; life insurance; time off/leave; well-being benefits.