Chief Medical Officer - Gene Therapy and Rare Disease

  • New Jersey
  • Permanent

Chief Medical Officer - Gene Therapy and Rare Disease

Our client is a small biotech company with promising gene therapy assets in preclinical development for rare genetic disorders. With funding for the next 2 years, they are now seeking an exceptional physician leader to serve as Chief Medical Officer and build out their full R&D function including clinical development, clinical operations, regulatory affairs, biometrics, medical affairs and drug safety. This will involve advancing the lead candidate into Phase I studies. This is a pivotal leadership role overseeing all R&D, clinical, medical and regulatory affairs for the company.


  • Provide strategic medical and scientific leadership across all R&D stages from preclinical through regulatory approval for gene therapy programs
  • Design and build out the full R&D organization including clinical development, clinical operations, regulatory affairs, biometrics, medical affairs and drug safety
  • Oversee design and execution of global clinical trials evaluating novel gene therapy candidates
  • Lead interactions with regulatory agencies on INDs, clinical trial design, submissions and product approvals
  • Develop R&D strategic plans, design study protocols, statistical analysis plans and regulatory submissions
  • Analyze and interpret preclinical/clinical data, lead data monitoring committees
  • Ensure highest standards of medical safety monitoring, pharmacovigilance and risk management
  • Identify and collaborate with leading gene therapy researchers, scientific advisors and clinical experts
  • Build and lead high-performing R&D teams across development disciplines and multiple sites
  • Represent the company as a scientific/medical expert at investor, partner and scientific conferences


  • M.D. or M.D./Ph.D. with 15+ years of experience leading R&D, clinical trials, regulatory approvals and product launches
  • Extensive expertise in gene therapy clinical development and R&D for rare diseases
  • Significant expertise with AAV vectors or other gene transfer/therapy technologies
  • Proven track record with successful regulatory authorities like FDA for breakthrough product approvals
  • Exceptional scientific/medical credibility with elite academic research experience
  • Outstanding leadership to inspire and build out a complete R&D organization
  • Strategic mindset with ability to drive integrated R&D plans from preclinical to commercial
  • Expertise in study design, data analysis, regulatory strategy and submissions
  • Entrepreneurial passion for bringing transformative rare disease therapies to patients
  • Prior experience building R&D teams/functions from the ground up is strongly preferred
Nicole Crowley Senior Consultant

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