Program Head, Gene Therapy and Rare Disease

P95 • ,

3d

About The Position

The Program Head, Gene Therapy and Rare Disease is a senior leadership role within the Neuroscience Business Unit, responsible for the end-to-end oversight, strategic direction, and growth of the Gene Therapy and Rare Disease portfolio. The role ensures successful delivery of complex global clinical programs for sponsors while driving operational excellence, profitability, and long-term client partnerships.

The position carries full accountability for portfolio performance, including delivery outcomes, financial performance, and sponsor satisfaction. It also leads the development and scaling of differentiated Cell & Gene Therapy (CGT) capabilities to support future growth and market leadership.

Requirements

• 12+ years of experience in clinical research within a CRO, biotech, or pharmaceutical environment.
• Extensive experience managing global clinical programs in Gene Therapy, Rare Disease, and/or Neuroscience.
• Demonstrated success with portfolio accountability, including financial ownership (P&L).
• Proven experience managing executive-level sponsor relationships.
• Experience supporting or leading RFPs, bid defenses, and commercial strategy.
• Deep knowledge of global regulatory requirements, including GCP, ICH, and regional regulations.
• Experience operating in global and matrixed environments.
• Strategic leadership within a CRO delivery model.
• Strong commercial and financial acumen.
• Ability to lead complex, global clinical programs.

Nice To Haves

• An advanced degree (PhD, MD, PharmD, or Master’s) in Life Sciences or a related field is preferred.

Responsibilities

• Provide executive oversight of the Gene Therapy and Rare Disease portfolio within the Neuroscience Business Unit.
• Serve as a senior escalation point for sponsor relationships, driving client satisfaction, retention, and repeat business.
• Lead CRO service delivery across programs, including study start-up, feasibility, enrollment strategy, and execution.
• Drive the development and expansion of Cell & Gene Therapy capabilities and differentiated service offerings.
• Own portfolio financial performance, including revenue, margins, forecasting, and resource optimization.
• Partner with Business Development and RFP teams to shape proposals, define execution strategies, and support bid defenses.
• Establish and expand indication-specific investigator networks for rare disease and gene therapy trials.
• Use data-driven approaches to improve enrollment performance, including feasibility analytics and screening optimization.
• Identify and execute strategic growth opportunities in Gene Therapy and Rare Disease aligned with sponsor demand and market trends.

What This Job Offers

• Job Type: Full-time
• Career Level: Senior
• Education Level: No Education Listed