CMO
Position Overview
The Chief Medical Officer (CMO) will be a key member of the executive team, responsible for defining and executing clinical strategy across the pipeline. This individual will lead the transition into early clinical development and help shape multiple programs built on a shared platform.
This role requires both strategic leadership and hands-on execution in an early-stage environment.
Key Responsibilities
Clinical Strategy
- Lead clinical strategy across programs
- Design and oversee early-phase trials
- Define endpoints for rare genetic diseases
- Align preclinical, clinical, and regulatory plans
Translational & Biomarkers
- Develop biomarkers for target engagement and efficacy
- Integrate translational data into clinical decisions
- Ensure strong mechanistic rationale
Regulatory
- Lead IND strategy and regulatory interactions
- Interface with FDA and global agencies
- Support orphan and expedited pathways
Pipeline & Leadership
- Prioritize programs and allocate resources
- Build and lead a lean clinical team
- Partner cross-functionally across research, regulatory, and CMC
External Engagement
- Engage KOLs and investigators
- Support investor and board communications
- Contribute to partnerships and strategy
Qualifications
Required
- MD or MD/PhD with clinical development experience
- Track record advancing programs into early trials
- Experience in rare disease, neurology, or related areas
- Strong clinical trial and regulatory expertise
- Strategic and hands-on leadership ability
PREFERRED
- Experience with RNA or platform-based therapeutics
- Background in translational medicine and biomarkers
- Experience in early-stage biotech environments
Why This Role
This is an opportunity to lead clinical development for a novel platform at a critical stage. The CMO will shape multiple programs advancing toward the clinic while building a high-impact function within a collaborative, mission-driven company focused on serious unmet medical needs.