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.

• 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.

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

• 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.

• 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.