CMO (, MA, United States)

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.