Original listing text, shown exactly as published by the company.
Responsibilities
- Develop and execute medical affairs strategies aligned with company goals, including evidence generation, scientific exchange, and medical input into cross-functional teams.
- Build and maintain strong relationships with HCPs, KOLs, and patient advocacy organizations in DMD, Becker muscular dystrophy (BMD), and related fields through local, national, and international meetings, 1:1 site visits, and advisory boards.
- Collaborate with the CMO to establish high-integrity, compliant processes for HCP and KOL engagement, ensuring adherence to regulatory guidelines (e.g., FDA, ICH).
- Create and refine the clinical scientific and value story for deramiocel through internal consultations, external expert input, and data analysis to support program progression.
- Lead scientific communications, including publications, congress presentations, and medical information responses, while overseeing vendors as needed.
- Provide medical expertise to support commercialization readiness activities, including training, payer interactions, and real-world evidence initiatives.
- Work cross-functionally with clinical development, regulatory affairs, commercial, and quality teams to drive business impact and team success.
- Identify opportunities for continuous improvement in medical affairs operations, fostering a culture of innovation and compliance.
- Monitor emerging scientific data, competitive landscape, and therapeutic trends in neurology, myology, cardiology, and rare diseases to inform strategy.
- Perform such other duties as may be assigned from time to time.
Requirements
- MD required, with experience in neurology, myology, cardiology, or a closely related field, and demonstrated clinical scientific competence.
- Deep experience and commitment to excellence in medical affairs, with a proven track record in biotechnology or pharmaceuticals.
- Experience working in a small company environment, thriving in dynamic, resource-constrained settings.
- Passionate "can-do" mentality with evidence of high emotional intelligence (EQ) and collaborative teamwork.
- Exceptional communication, presentation, and interpersonal skills to engage stakeholders at all levels.
- Strong understanding of regulatory compliance, including cGMP, ICH, and FDA guidelines.
- Ability to work independently, multi-task, and meet aggressive deadlines in a fast-paced environment.
- Willingness to travel up to 30-40% for meetings, site visits, and conferences.
Preferred Qualifications
- Previous experience with DMD, BMD, or other muscle or cardiomyopathic indications.
- Experience with pre-launch or commercialization activities in a Medical Affairs capacity.
- Experience with complex cell or gene therapy (ATMP) products.
Work Environment and Physical Demands
- Professional office (San Diego) or remote work environment.
- Prolonged periods of computer use for planning, documentation, and reporting.
- Occasional travel may be required for meetings, site visits, and stakeholder engagement.
- Ability to lift office materials up to 20 pounds.
