Investigator-Initiated Studies

We are fully committed to developing precision genetic medicine aimed at improving and extending the lives of patients with rare diseases. As part of that commitment, we recognize the potential of independent research to enhance understanding and answer important medical and scientific questions regarding our core therapeutic areas of interest.

Click here to learn about some of the rare diseases we’re researching.

IIS Web Graphics_IIS Overview

About the Program

Investigator-initiated studies (IIS) are independent research studies or investigations that are initiated, conducted, and sponsored by an investigator.

We would like to hear from individual researchers, institutions, or groups interested in requesting funding or material for a clinical or non-clinical IIS in rare neuromuscular diseases.

Applications are accepted on a rolling basis and are reviewed monthly. Our team will review your request and provide feedback within 90 days of submission.

See our FAQ for additional information.

Application Process

Submit a Proposal

To submit a concept form, please connect with a Sarepta Medical Affairs team member or email [email protected].

To submit a proposal, please chose from one of the two options below.

Submit Clinical Proposal Submit Non-clinical Proposal

Frequently Asked Questions

  • What is the difference between a concept and proposal review?

    A concept is an optional 1–2-page high level summary used for Sarepta’s review and initial recommendation. If Sarepta is interested in the study concept, investigators must submit an application and full study proposal to be considered for funding. Proposals must include study background, objectives, outcome measures, methodology/study design, study duration, budget, milestones, and timelines.

  • Do I have to submit a concept form?

    No, submitting a concept form is an optional step which allows Sarepta to make an initial recommendation. If the review committee decides the study is in scope, investigators must submit an application and full proposal through Sarepta’s Web Portal to be considered for funding. Final assessment is based on the application and full proposal.

  • What are the minimum requirements of a proposal?

    Proposals must include study background, objectives, outcome measures, methodology/study design, study duration, budget, milestones, and timelines.

  • Do I need to submit any additional documentation with my proposal?

    Yes, a detailed budget, CV and the institution’s official tax documentation are needed.

  • How do I submit a concept?

    To submit a concept form please connect with a Sarepta Medical Affairs team member or email [email protected]. To submit a full proposal, please click here.

  • What types of studies are eligible?

    Clinical studies (including observational studies) and non-clinical studies in rare neuromuscular diseases are eligible. Please note, proposals to develop databases or registries without defined research objectives or those that are not designed to address a valid scientific question are typically not supported by the IIS program. If you have any questions regarding study eligibility, please email [email protected] or connect with a Sarepta Medical Affairs team member. 

    For requests related to compassionate use, please refer to our Compassionate Use Policy for more information.

  • How long is the review process?

    Applications are reviewed monthly, and feedback is provided within 90 days.

  • How are proposals evaluated?

    Sarepta’s subject matter experts and IIS committee critically review proposals and evaluate them based on scientific merit and alignment with Sarepta’s strategic areas of interest.

  • Is there a limit on funding support offered by Sarepta?

    The amount of funding provided to each IIS is dependent on the study type. Each study is evaluated independently, and all funding requests are subject to a fair market value assessment. Purchase of capital investments (e.g., equipment, laptops, software) are not supported.

  • How are payments made?

    Approved proposals are based on milestone payments. If you have any additional questions regarding payment, please contact [email protected].

  • What are my responsibilities if my study is approved?

    Investigators will be responsible for the following (as applicable),  including but not limited to:

    • Developing a research protocol and obtaining IRB approval
    • Maintaining rigorous standards for research integrity and data quality
    • Providing regular study updates to the Sarepta team and a written report upon study completion
    • Committing to disclosing and disseminating the findings in an appropriate, transparent, and timely manner in accordance with agreed timelines
    • Ensuring compliance with all necessary safety reporting requirements by Sarepta and local/national health authorities

Questions?

If you have any questions about the IIS program, please contact [email protected] or your Sarepta Medical Science Liaison (MSL).