Director, Clinical Development – Gene Therapy

This is a fully remote position, open to applicants in United States.

📋 Description

• Provide support for clinical development across multiple gene therapy programs at various stages of the drug development lifecycle.

• Assist with clinical development for ETX301 and oversee pharmacovigilance and medical monitoring for ETX101.

• Aid in investigator management and collaborate on regulatory interactions and outreach to patient advocacy groups.

• Contribute expertise to content development and publications.

• Support strategy for clinical development targeting chronic neuropathic pain.

• Lead efforts related to Phase 1–4 clinical trials, which includes authoring and reviewing protocols, investigator brochures, clinical study reports, and managing CROs.

• Define, plan, and offer medical monitoring support for ongoing and completed studies related to Dravet syndrome and chronic neuropathic pain.

• Review adverse events, monitor real-time safety data, prepare analyses and clinical insights for regulatory submissions, and lead data interpretation alongside pharmacovigilance CRO partners.

• Act as the clinical representative for Data Monitoring Committee safety reviews, ensuring ongoing safety monitoring.

• Assess complex screening data and verify participant eligibility with the Eligibility Review Committee.

• Foster relationships with KOLs, principal investigators, pediatric clinical trial networks, outcomes experts, and patient advocacy organizations.

• Lead investigator meetings and site initiation visits, collaborating with Clinical Operations and Medical Affairs to enhance enrollment.

• Define roles, responsibilities, and performance expectations for direct reports; provide feedback and identify opportunities for growth.

• Work collaboratively with internal clinical, regulatory, scientific, and operational teams as well as external experts, investigators, and government entities.

⛳️ Requirements

• An MD, DO, or equivalent international medical degree is required.

• A minimum of 3 years of clinical development experience in biotech/pharma is necessary for the Director level.

• Equivalent experience as an academic physician-scientist leading clinical trials as a principal investigator will also be considered.

• Proven experience in designing and executing clinical trials, with strong vendor management capabilities.

• Ability to communicate effectively with scientists, thought leaders, investigators, and executive management.

• A demonstrated history in medical monitoring, assessing potential dose-limiting toxicities, pharmacovigilance, and GCP/ICH regulatory compliance.

• Experience in overseeing protocol-mandated prophylactic immunomodulation regimens is highly desirable.

• Experience in managing direct reports, leading cross-functional project teams, and establishing collaborative relationships with key opinion leaders.

• Exceptional oral and written communication skills, including the ability to translate complex clinical data into presentations, regulatory documents, and peer-reviewed publications.

• Strong collaboration and interpersonal skills with internal stakeholders across Regulatory Affairs, Medical Affairs, Clinical Operations, Biometrics, Research, and Translational Science.

• Proficient computer skills, including MS Office Suite, electronic data capture systems like RAVE and InForm, data analysis software, and data presentation tools.

• Willingness to travel up to 25%, including potential international travel.

• Board certification or eligibility in relevant medical specialties is preferred.

• Specific experience in gene therapies, rare diseases, epilepsy, chronic pain management, medical monitoring, and pharmacovigilance is preferred.

• Experience with clinical-regulatory strategies and/or industry-academic collaborations is preferred.

• Experience providing clinical input for regulatory submissions and engaging with global health authorities is preferred.

• Knowledge of AAV gene or cell therapy risks, immunogenicity, dorsal root ganglionopathy, hepatotoxicity, and long-term follow-up requirements is preferred.

🏝️ Benefits

• Comprehensive benefits package, including competitive employer premium contributions.

• Meaningful stock option grants.

• PTO, sick leave, and holiday pay.

• Generous Parental Leave program.

• Pre-tax medical and dependent care programs.

• STD, LTD, Life, and AD&D insurance.

• Opportunities for professional development.

• Team-building events.

• Fully stocked micro-kitchen.

• Fitness center located at the South San Francisco office.

• Discretionary annual bonus.

• Discretionary stock-based long-term incentives.