Chief Medical Officer (CMO)
Location: Greater Boston Area, Remote
Stage: Venture-backed, Preclinical to Early Clinical
Company Overview
We are a venture-backed biotechnology company developing a novel class of RNA-based therapeutics designed to address the genetic drivers of disease. Our platform restores functional protein expression in conditions caused by defined mutations, offering a differentiated, potentially disease-modifying approach beyond small molecules, biologics, and gene editing.
We are building a pipeline across rare and severe genetic disorders with high unmet need, including neurological, neurodevelopmental, neuromuscular, and cardiometabolic diseases. The platform is broadly applicable, enabling expansion into additional indications.
With strong investor backing and growing preclinical validation, the company is approaching a key inflection point as programs advance toward IND-enabling studies and first-in-human trials.
Position Overview
The Chief Medical Officer 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.
The CMO will work cross-functionally to translate preclinical insights into clinical hypotheses and well-designed studies. 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.
