Cure Rare Disease · 18 hours ago
SVP Clinical Development
United States
Full-time
Remote
Director/Executive
$320K/yr - $350K/yr
7+ years expCure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing genetic medicines for rare and ultra-rare diseases. They are seeking a visionary SVP Clinical Development to lead clinical and medical strategies, oversee the transition of therapeutics into clinical trials, and ensure alignment with patient needs through strategic engagement and scientific communication.
BiotechnologyHealth CareNon ProfitWellness
Hiring Manager
Rich Horgan
Responsibilities
Pipeline Oversight: Together with the CEO, CSO and regulatory lead, you will manage the continued development of CRDs therapeutic pipeline. You will direct the clinical development roadmap for CRD’s portfolio of rare and ultra-rare therapeutics, moving programs from "bench to bedside" with a focus on rare and ultra-rare cohorts
Trial Design: Design robust, adaptive clinical trial protocols, investigator’s brochure and IRB submissions/negotiations suitable for rare and ultra-rare patient populations, ensuring valid endpoints and data integrity
Patient Selection: Lead the rigorous evaluation of potential patient candidates, analyzing genetic reports and natural history data to determine suitability for CRD’s therapeutic modalities (gene editing vs. replacement)
Team building: Identify, in collaboration with leadership team, future clinical hires such as clinical trial operations as needed
Grant Authorship: Lead the medical/clinical writing for major grant applications (e.g., NIH, FDA Orphan Products Grants, private foundations, and industry partnerships)
Proposal Development: Translate nonclinical data and clinical trial designs into compelling, evidence-based narratives that demonstrate scientific viability and patient impact to reviewers
FDA Interaction: Serve as the key liaison with regulatory bodies (FDA, EMA) for clinical topics. In partnership with CRD’s regulatory lead, drive the preparation and defense of IND (Investigational New Drug) applications, BLA applications, INTERACT meetings, and pre-IND consultations
FDA designations: Support applications for regulatory designations including ODD, RMAT, Fast Track, Breakthrough Therapy and Priority Review
Safety Oversight: Establish and chair the Data Safety Monitoring Board (DSMB) and internal safety review committees; ensure strict adherence to GCP (Good Clinical Practice) and ethical standards
Risk Management: Proactively identify clinical risks in novel gene therapies and develop mitigation strategies to protect patient safety above all else
Scientific Advisory Board (SAB): Recruit and manage the clinical SAB, fostering collaboration among leading academic researchers and clinicians
Thought Leadership: Represent Cure Rare Disease at medical conferences, in peer-reviewed publications, and within the rare disease community to elevate the organization's scientific standing
Patient Engagement: Act as an empathetic and clear communicator to patient advocacy organizations and families, explaining complex medical risks, trial processes, and realistic outcomes for experimental therapies
Qualification
Required
M.D. or M.D./Ph.D. required
7+ years of leadership experience in clinical development within the biotech, pharmaceutical, or academic research sectors
Direct experience with gene therapy, precision medicine, or rare disease drug development is essential
Experience in authoring and contribution to competitive grants (NIH R01, SBIR/STTR, CIRM etc.)
A solid understanding of FDA regulatory pathways for rare diseases
Experience of writing and defending regulatory submissions (e.g. preIND, IND, accelerated approval, orphan drug designation)
Ability to balance scientific rigor with deep compassion for families facing fatal diagnoses
Comfortable working in a fast-paced, 'startup-like' non-profit environment where wearing multiple hats is the norm
Exceptional writer and speaker capable of distilling complex science for both expert reviewers and lay audiences
Preferred
Board certification in Neurology, Genetics, Pediatrics, or a related field is highly preferred
Benefits
Health benefits
Performance bonus
Company
Cure Rare Disease
Cure Rare Disease is a biotechnology company.
2-10 employees
Funding
Current Stage
Early StageTotal Funding
$13.04MKey Investors
California Institute for Regenerative MedicineMuscular Dystrophy Association
2025-12-11Grant· $7.35M
2025-02-06Grant· $5.69M
2023-02-16Grant
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