Director, Clinical Science Lead

Overview

Work at ROCKET PHARMA and help cure rare diseases!

 

Rocket Pharma is a fully integrated, clinical-stage company advancing gene therapies with curative potential for multiple rare childhood diseases. Rocket places enormous value on people and considers team member progress and well-being to be as important as the progress of its pipeline. We are looking for hands-on team players who enjoy collaborating with colleagues. We share a passion for seeking gene therapy cures for devastating diseases and making a difference for patients. This is an opportunity to become an integral part of a small, highly productive team and to help grow an entrepreneurial, scientifically driven organization that is increasingly recognized as a leading biotechnology innovator. In addition to a competitive compensation package featuring a generous 401K match and stock options, the position includes excellent health benefits.

Position Summary

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.

Responsibilities

• 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

Qualifications

• 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 tr