Organization: Cure Rare Disease (CRD)

Location: Remote

Reports to: Founder & Chief Executive Officer

About Cure Rare Disease

Cure Rare Disease (CRD) is a non-profit biotechnology organization with a mission to enable and finance the development of life-saving genetic medicines for rare and ultra-rare patient populations previously deemed "too rare to treat." We are disrupting the traditional drug development model by building a collaborative ecosystem of world-renowned researchers, clinicians, and donors. Our focus is on developing customized therapeutics (CRISPR, AAV, ASO) for neuromuscular and neurodegenerative diseases that commercial pharma has left behind.

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Role Overview

We are seeking a visionary, hands-on, and scientifically rigorous SVP Clinical Development to lead our clinical and medical strategies. The SVP Clinical Development will serve as the primary medical voice of the organization, overseeing the transition of therapeutics from preclinical research into first-in-human clinical trials and beyond.

This role requires a hybrid of clinical leadership and scientific communication. You will guide patient safety and regulatory strategy and also engage with patient organizations to ensure alignment of CRDs programs with patient needs. In conjunction with our CSO you will play a pivotal role in securing the resources necessary to fuel our mission. You will leverage your deep medical expertise to author and lead high-level grant proposals, ensuring our groundbreaking science is backed by sustainable funding.

Central to CRDs mission is the acceleration of drug development. The SVP Clinical Development will engage with the CSO, regulatory lead and collaborators to anticipate potential clinical liabilities and identify de-risking strategies. Ensuring that candidate development is as rapid as possible, while maintaining patient safety as the central focus.

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Key Responsibilities

1. Clinical Development & Strategy

        1. 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.

       2. 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.

       3. 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).

       4. Team building: Identify, in collaboration with leadership team, future clinical hires such as clinical trial operations as needed.

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2. Scientific Grant Writing & Funding Strategy

• 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.

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3. Regulatory Affairs & Compliance

• 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.

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4. Medical Leadership & Stakeholder Engagement

• Scientific Advisory (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.

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Qualifications

• Education: M.D. or M.D./Ph.D. required. Board certification in Neurology, Genetics, Pediatrics, or a related field is highly preferred.

• Experience:

    o 7+ years of leadership experience in clinical development within the biotech, pharmaceutical, or academic research sectors.

   o Direct experience with gene therapy, precision medicine, or rare disease drug development is essential.

    o Experience in authoring and contribution to competitive grants (NIH R01, SBIR/STTR, CIRM etc.).

• Regulatory Knowledge: 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).

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Soft Skills:

o Empathetic Leader: Ability to balance scientific rigor with deep compassion for families facing fatal diagnoses.

o Agility: Comfortable working in a fast-paced, "startup-like" non-profit environment where wearing multiple hats is the norm.

o Communication: Exceptional writer and speaker capable of distilling complex science for both expert reviewers and lay audiences.

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Why Join Cure Rare Disease?

• Direct Impact: You will not just be managing a portfolio; you will be building therapies for children who have no other hope.

Innovation: Work at the bleeding edge of genetic medicine, setting precedents for how rare and ultra-rare therapies are regulated and delivered.

•Culture: Join a passionate, mission-driven team that values transparency, urgency, and scientific excellence.

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Compensation:

• $320,000 - $350,000 per year

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Interest:

•Please email cover letter and resume to rich@cureRD.org

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