The Director, Clinical Science Lead works in close collaboration with cross-functional medical, clinical operations, regulatory and manufacturing team members, academic partners, and external vendors to enable optimal study design, conduct and analysis for Rocket’s transformative gene therapy programs in hematologic and cardiovascular disorders. This position reports through the Clinical Development function and provides scientific input during protocol development, oversight of study conduct and data acquisition, and interpretation of study results. The Director, Clinical Science Lead directly manages Clinical Scientists across LV and AAV programs, delineating and refining their roles on respective development teams, identifying opportunities for growth and contribution, and defines best practices for integrated contributions in concert with medical directors, clinical operations, biometrics, medical writing, pharmacovigilance, natural history and medical affairs colleagues.

• Provide scientific input during protocol development, study design, oversight of study conduct, interpretation of study results through the final study report
• Interface with a cross-functional team including Clinical Operations, Data Management, Statistics, Drug Safety, Regulatory, Project Management, Medical Affairs and CMC to execute and deliver program objectives
• Contribute to protocol feasibility assessments, site identification, and vendor evaluation
• Collaborate with Clinical Operations to review vendor, CRO and site scope of work, study budget, and plans or manuals related to study data, IVRS, central labs, etc.
• Support preparation of abstracts, manuscripts, presentations, and materials for external meetings as related to the assigned protocol(s) and/or clinical program
• Assist in writing documents to support study conduct and regulatory submissions, including protocols, investigator brochures, informed consent forms, training documents and clinical study reports; lead authorship for laboratory and pharmacy manuals
• Develop internal and external training materials and training presentations, including site initiation visits
• Summarize potential safety and efficacy trends in clinical trial data, for preparation of interim and final reports of clinical trial data for study documents (including CSRs, IBs and DSURs)
• Interact with academic partners, collaborators, advisory board members, etc. as appropriate
• Support planning and conduct of study committee meetings (e.g. IDSMC), investigator meetings, and advisory boards, including contribution to presentations and minutes
• Management and mentorship of Clinical Scientists, including identification of responsibilities for which Clinical Scientists assume a lead or contributory role, and identify growth opportunities for Clinical Scientists within each project team
• Defines best practices for Clinical Scientist contributions to all aspects of strategic development, protocol design and conduct, development of key supporting materials, study site and vendor interactions, and data analysis/presentation
• Participates in leadership meetings within the Chief Medical Officer and Clinical Development organization to identify strategic and operational challenges and potential solutions and collaboration opportunities

• Advanced Degree in Life Sciences (i.e. DNP, APRN, PA or related medical field, PharmD, PhD) with 5 years experience; will consider Master’s degree with minimum of 8 years related experience, or Bachelor’s degree with 10 years of related experience, or equivalent experience
• Experience with gene therapy/rare disease trials and familiarity with frequently used evaluations
• Knowledge of registrational clinical trial design, statistics, and data review tools
• Experience reviewing and analyzing clinical trial data to identify potential safety and efficacy trends
• Significant experience in the development of protocols, case report forms, informed consent documents, and study initiation/monitoring activities
• Ability to share scientific data effectively across functions and through presentations
• Ability to collaborate across functions and job levels within the company, as well as with external vendors and academic partners
• Track record of management/mentorship in clinical development organization
• Attention to detail with excellent time management and organizational skills, and flexible attitude with respect to work assignments and new learning
• Self-starter and who functions well without extensive supervision
• Comfortable in a fast-paced small company environment with the ability to manage a variety of projects simultaneously and handle rapidly changing information
• Expert MS Office skills with a specific focus on word processing, formatting tables, spreadsheets, presentations, graphics, slides, and templates, in addition to Adobe Acrobat
• Knowledge of ICH Guidelines, including GCP
• Awareness of global regulatory and pharmacovigilance environments
• Preferred experience in the following areas:
  • Gene therapy, cell therapy or rare disease experience
  • Ability to function independently in data review to identify discrepancies in clinical trial data and to write queries from a medical perspective.
  • Familiarity with Medidata, RAVE, J-review or other clinical databases or other data review tools (patient profiles, listings).
• Limited Travel required, anticipated 5-10% overall.

A diverse workforce fosters innovation and strengthens Rocket’s business. We ensure equal opportunity without discrimination or harassment in the workplace on the basis of gender, race, color, religion, national origin, age, physical or mental disability, pregnancy, citizenship, status as a protected veteran, marital status, sexual orientation, gender identity and expression, genetic information, or any other characteristic protected by applicable local, state, and federal laws.