Position Summary:

The Sr. Medical Director, Clinical Sciences, will work closely with the Head of Clinical Sciences, to provide medical leadership and operational oversight for clinical development programs in hemoglobinopathies, specifically working on an ongoing registration-directed trial in children with sickle cell disease (SCD), the initiation of new trial(s) to evaluate a new manufacturing process and treatment of SCD patients with a history of stroke, and improving the efficiency of ongoing registry and long-term follow-up trials. This role will collaborate cross-functionally with Clinical Operations, Regulatory Affairs, Translational Medicine, Medical Affairs and external stakeholders to ensure the successful execution of clinical trials, analysis and reporting of clinical trial data, and advancement of gene therapy programs.

This is a full-time hybrid position (3 days a week) in our Somerville – Assembly Row, Massachusetts office. 

Key Responsibilities:

• Clinical Development Strategy:
• Lead the execution of clinical trials for gene therapies targeting hemoglobinopathies.
• Provide medical input into clinical protocols, investigator brochures, and regulatory submissions.
• Medical Oversight:
• Serve as medical monitor for ongoing clinical trials, ensuring patient safety and data integrity.
• Review and interpret clinical data, including safety and efficacy endpoints.
• Cross-Functional Collaboration:
• Partner with Regulatory Affairs to support interactions with health authorities.
• Collaborate with Translational Medicine and Research teams to align clinical strategy with scientific insights.
• External Engagement:
• Represent the company in interactions with key opinion leaders, investigators, and advisory boards.
• Collaborate with Medical Affairs to support Scientific Communications and Medical Information and Education.
• Compliance & Ethics:
• Ensure all clinical activities are conducted in accordance with GCP, ICH guidelines, and applicable regulations.

Qualifications:

• MD or MD/PhD, preferably with experience in Pediatrics, Stem Cell Transplantation, Hematology or a related field.
• Minimum of 5 years of experience in clinical development, preferably with experience in rare diseases or gene therapy.
• Understanding of hemoglobinopathies, including sickle cell disease and beta-thalassemia.
• Prior experience as a medical monitor and in regulatory submissions (IND, BLA).
• Strong leadership, communication, and strategic thinking skills.
• Ability to work independently in a fast-paced, dynamic environment.

Preferred Experience:

• Experience in biotech industry.
• Familiarity with lentiviral vector-based gene therapy
• Prior involvement in pivotal trials or product approvals.