Vice President – Clinical Development – Gene Therapy

Vice President – Clinical Development – Gene Therapy

Requirements

• MD required, preferably with Board certification, with specialization in rare diseases, neurology, or neuromuscular diseases is strongly preferred.
• 12+ years clinical development experience in the pharmaceutical or biotech industry with direct experience in Phase 2-4 clinical development, particularly in complex or rare diseases.
• Deep experience within the gene therapy realm to include knowledge of multiple therapeutic areas.
• Demonstrated success in building and maintaining strong relationships with Key Opinion Leaders (KOLs) and external experts to advance gene therapy assets.
• Extensive experience developing and submitting complex NDAs and BLAs is required.
• Previous direct people management experience is paramount for this role.
• Strong working knowledge of Good Clinical Practice (GCP), scientific and clinical methodology, clinical trial design, project management, and the regulatory landscape specific to gene therapy or rare disease clinical studies.
• Demonstrated leadership presence, with the ability to work effectively with clinical, regulatory, and scientific leaders both within Insmed and externally.
• A problem-solver and collaborative team player who actively encourages and values the contributions of others.
• Strong patient focus with a passion for advancing medical treatments through the development of innovative gene therapies. Commitment to making a meaningful impact on patient care.
• Excellent communication skills, with an emphasis on transparency, clarity, and precision. Highly organized with strong attention to detail, accuracy, and the ability to convey complex scientific concepts clearly.
• Demonstrated ability to work effectively both independently and as part of a team. Strong internal-facing leadership and interpersonal skills with a high degree of professionalism, maturity, and the ability to navigate complex gene therapy development environments.
• Strong analytical skills with the ability to interpret complex data sets and make informed decisions.
• Experience in vendor management is a plus, particularly in the context of outsourced gene therapy or rare disease development services and collaborations.
• Willingness to travel up to 35%, both domestically and internationally, with potential periods requiring up to 50% or more travel, particularly for gene therapy trial oversight and KOL engagements.
• Must exemplify Insmed’s values of Collaboration, Accountability, Passion, Respect, and Integrity.

Responsibilities

• Provide medical/scientific expertise by working closely with Clinical Scientists and Clinical Operations teams throughout the lifecycle of gene therapy clinical studies, from pre-study planning to post-study analysis. Proactively identify and resolve study design issues that may arise during gene therapy trials.
• Co-lead a global study team, alongside Program Management, to advance gene therapy assets, particularly through Phase II and beyond. Collaborate effectively with cross-functional teams such as Data Management, Biostatistics, and Regulatory Affairs, to ensure seamless execution of the gene therapy development plan.
• Offer medical expertise across functions, including collaborating with Drug Safety and Pharmacovigilance to monitor safety signals and manage gene therapy-related adverse events.
• Support the preparation and presentation of scientific materials, such as abstracts, manuscripts, and conference presentations, as well as Advisory Board materials relevant to gene therapy programs.
• Present protocols and other materials at Investigator Meetings, training sessions, and other internal or external forums as needed, particularly related to gene therapy studies.
• Provide medical guidance during data reviews, interim analyses, and blinded data assessments for gene therapy trials.
• Review and provide feedback on statistical analyses and collaborate with Data Management teams on the development of eCRFs, edit checks, coding, and other data processes necessary for gene therapy studies.
• Stay current on scientific and medical advancements in gene therapy and maintain relationships with external experts. Represent Insmed in external groups and professional organizations focused on gene therapy.
• Contribute to the strategic development of both early and late-stage gene therapy clinical programs, ensuring alignment with corporate objectives and regulatory requirements.
• Participate in due diligence activities as needed, contributing medical and scientific expertise during the evaluation of investigational agents and/or platforms.

Preferred Qualifications

• Experience in vendor management is a plus, particularly in the context of outsourced gene therapy or rare disease development services and collaborations.