Role Overview for Chief Medical Officer (CMO) with a Rare Disease Biotech – San Diego, CA
JRG Partners has been exclusively retained to lead the search for a transformative Chief Medical Officer (CMO) for a well-funded, clinical-stage biotechnology company based in the vibrant life sciences hub of San Diego, CA. This pioneering organization is dedicated to developing novel, life-changing therapies for patients suffering from devastating rare diseases with significant unmet medical needs. This is a unique opportunity to join a passionate and science-driven executive team at a pivotal moment, shaping the future of the company and making a profound impact on patient lives.
As the Chief Medical Officer, you will be the senior-most clinical leader in the organization, providing the strategic vision and hands-on leadership for all aspects of clinical development, medical affairs, and drug safety. Reporting directly to the Chief Executive Officer (CEO), you will serve as a critical member of the executive leadership team, contributing to the overall corporate strategy, investor relations, and business development activities. The ideal candidate is a strategic, innovative, and execution-oriented physician-scientist with a proven track record of successfully advancing therapies through the clinic to regulatory approval, specifically within the challenging and rewarding field of rare disease. You will be responsible for building and mentoring a world-class clinical organization, fostering a culture of scientific excellence, urgency, and unwavering patient focus. This role requires a leader who can seamlessly blend deep scientific and medical expertise with astute business acumen, capable of navigating the complex regulatory landscapes and inspiring confidence in internal teams, external partners, and the investment community.
This position is not merely about oversight; it is about building, innovating, and leading from the front. You will be the primary medical voice of the company, representing its clinical programs to regulatory agencies, key opinion leaders (KOLs), patient advocacy groups, and the broader medical community. Your leadership will be instrumental in translating groundbreaking science into approved medicines that offer new hope to patients and their families. If you are a visionary medical leader driven by a passion to conquer rare diseases and build a lasting legacy, this is your defining career opportunity.
Key Responsibilities of Chief Medical Officer (CMO) with a Rare Disease Biotech – San Diego, CA
Clinical Development Strategy & Execution
Provide strategic leadership for the entire clinical development portfolio. Design, implement, and oversee innovative and efficient clinical trials (Phase I-IV), employing adaptive designs, novel endpoints, and biomarker strategies appropriate for small patient populations. Ensure all clinical studies are conducted with the highest standards of quality, ethics, and regulatory compliance (GCP).
Regulatory Leadership
Serve as the primary medical liaison with global regulatory authorities, including the FDA, EMA, and others. Lead the development and execution of the global regulatory strategy, overseeing the preparation and submission of key documents such as INDs, CTAs, NDAs, BLAs, and orphan drug applications. Proactively navigate complex regulatory challenges to achieve timely approvals.
Medical Affairs & Scientific Communications
Build and lead a strategic, global Medical Affairs function. Develop and execute robust plans for KOL engagement, medical advisory boards, scientific publications, and medical education to support the company’s products and pipeline. Ensure the dissemination of accurate and compelling clinical data to the medical community.
Pharmacovigilance & Patient Safety
Establish and maintain a comprehensive global pharmacovigilance and risk management system. You will be ultimately responsible for monitoring, evaluating, and reporting all safety data, ensuring patient safety remains the top priority across all clinical programs.
Executive Leadership & Team Building
As a key member of the executive team, contribute to corporate strategy, long-range planning, fundraising, and business development initiatives. Recruit, mentor, and lead a high-performing team across clinical development, clinical operations, medical affairs, and drug safety. Foster a collaborative, inclusive, and results-oriented culture.
External Representation & Stakeholder Engagement
Act as the company’s lead medical spokesperson at scientific conferences, investor meetings, and public forums. Build and maintain strong relationships with leading clinical investigators, academic institutions, patient advocacy groups, and other key external stakeholders.
Budget and Resource Management
Develop and manage the annual budget for all clinical and medical functions, ensuring optimal allocation of resources to achieve corporate goals. Oversee the selection and management of contract research organizations (CROs), and other external vendors to ensure high-quality, on-time, and on-budget delivery of clinical programs.
Requirements for the Chief Medical Officer (CMO) with a Rare Disease Biotech – San Diego, CA
Educational Background
An M.D. is required. An advanced degree (Ph.D.) or M.B.A. is highly desirable. Board certification in a relevant medical specialty (e.g., Neurology, Genetics, Pediatrics, Hematology/Oncology) is a significant plus.
Professional Experience
A minimum of 15 years of progressive experience in clinical development within the pharmaceutical or biotechnology industry. At least 5-7 years of experience in a senior leadership capacity (e.g., Vice President, Senior Vice President, or CMO level) with a proven track record of managing and mentoring teams.
Rare Disease Expertise
Deep and direct experience in the clinical development of therapies for rare or orphan diseases is essential. You must have a strong understanding of the unique challenges and opportunities in this space, including trial design for small populations, patient advocacy engagement, and navigating orphan drug regulatory pathways.
Regulatory Success
Demonstrated success in leading programs through all phases of clinical development, culminating in successful marketing authorizations (NDA/BLA/MAA). Extensive, hands-on experience interacting with the FDA and EMA is mandatory.
Leadership Acumen
Proven ability to build, lead, and inspire high-performing, cross-functional teams in a fast-paced, entrepreneurial environment. Must possess exceptional strategic thinking, decision-making, and problem-solving skills.
Communication Skills
Outstanding written and verbal communication skills. Must be capable of effectively articulating the company’s clinical and scientific strategy to a wide range of audiences, from internal teams and board members to investors and KOLs.
Personal Attributes
A high degree of integrity, a strong work ethic, and a clear passion for the company’s mission. Must be adaptable, resilient, and comfortable with the ambiguity inherent in a dynamic biotech setting. A collaborative spirit and the ability to influence across the organization are critical. The candidate must be based in or willing to relocate to the San Diego, CA area.
Benefits & Perks Offered
Our client is committed to attracting and retaining top talent by offering a highly competitive and comprehensive compensation and benefits package. This includes:
Executive Compensation
A competitive base salary, a significant annual performance-based bonus, and a substantial equity package (stock options) providing a meaningful stake in the company’s success.
Comprehensive Health & Wellness
Premium medical, dental, and vision insurance for you and your eligible dependents. A robust wellness program and employee assistance program are also provided.
Retirement Savings
A 401(k) retirement plan with a generous company matching contribution.
Work-Life Balance
A generous paid time off (PTO) policy, including vacation, sick leave, and company holidays, to support a healthy work-life integration.
Relocation Support
A comprehensive relocation package is available for the successful candidate and their family.
Professional Growth
Ample opportunities for professional development, including support for attending key scientific conferences and continuing medical education.
Impactful Mission
The invaluable opportunity to work alongside a world-class team on a mission to deliver transformative therapies to patients with profound unmet needs.
How to Apply
JRG Partners is proud to be the exclusive recruitment partner for this confidential and critical search. We are seeking a truly exceptional medical leader who is ready to take on the challenge of a lifetime. If you are a visionary physician-scientist with a passion for rare diseases and you meet the qualifications outlined above, we invite you to begin a confidential discussion with us.
To apply, please submit your curriculum vitae (CV) and a compelling cover letter outlining your suitability for the role through our secure portal. All inquiries and applications will be handled with the utmost discretion. To learn more about our dedicated expertise in recruiting leaders for innovative companies, please visit our Life Sciences Executive Search Practice page.

