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Regenerative Therapies for Neuromuscular Targets, Spring 2021

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The goal of this request for proposals is to promote research with the potential to rapidly transform basic and translational regenerative medicine into treatments for SMA and other diseases.

  • With support from the Spinal Muscular Atrophy (SMA) Foundation, Stanford’s Innovative Medicines Accelerator (IMA) is seeking proposals from Stanford faculty to address unmet needs in the drug development pipeline for SMA and other devastating neuromuscular diseases.
  • The SMA Foundation is pursuing regenerative and cell-based therapies that will “rebuild” the nervous system and muscle that will not only impact SMA but other indications such as neurodegenerative diseases, muscular dystrophies, cachexia, and aging.
  • Read more about the SMA Foundation’s recently announced $60M investment in regenerative medicine.
  • The Foundation’s goal is to seed promising science at Stanford with awards up to $300,000 with the expectation that these projects will lead to new therapies and/or companies.

The RFP provides a unique opportunity to work with an experienced team with a track record for accelerating therapeutics development.

  • Since its inception, the SMA Foundation has funded over $150 million in research and therapeutics development and collaborated with leading academic medical centers (including Stanford), the federal government, contract research organizations, and many large pharmaceutical companies throughout the world.
  • The efforts of the SMA Foundation have led to the approval of three FDA approved drugs for SMA that address the genetic cause of the disease. Now, the Foundation is exploring regenerative medicine strategies that can further help SMA and other neuromuscular disease patients.  In addition to funding, the SMA Foundation can provide its valuable research tools, expertise, and connections to resources for drug development, all of which will help expedite the selected projects’ progress to the clinic and to patients; the SMA Foundation can also foster partnerships between funded faculty and CROs or pharmaceutical companies.

SMA Foundation

Read More About the SMA Treatment Landscape

Please log into your Stanford Box or Stanford Medicine Box account to access the linked materials.

Background Information from SMAF

Support Provided: Selected projects will receive a university research award of up to $300,000 for one year. Please note that award recipients will receive the entire amount funded, as overhead costs will be covered separately. If a project achieves its goal and makes significant progress towards a therapy, robust additional funding and resources for preclinical development may be awarded.

Deadline: All application materials must be received by 5pm on April 30th, 2021.

Eligibility: All Stanford faculty with PI eligibility are welcome to apply. Previous research experience in SMA is not required.

Application Guidelines: Submit one PDF file containing the following materials in the order indicated below. All documents should be single-spaced, Arial 11-point font with 1-inch margins.

  1. Title page (1 page)
    •  “SMA 2021 Request for Proposals”
    • Project title
    • Investigator(s): Name, department, address, phone number, email address 
    • Application summary (150 words) – Provide a concise statement of the Significance and the Approach of the proposed project. Successful proposals will define a one-year milestone that, if achieved, will justify more intensive drug prototyping efforts.
  2. Proposal (3 pages maximum)
    • Describe the therapeutic hypothesis and its supporting evidence. If a therapeutic target or pathway has already been identified, provide a rationale for its choice. If the goal of the project is to identify a druggable target/pathway, explain how this objective will be achieved. If the goal is to engineer a cellular therapy, describe the characteristics of a drug prototype, and how they will be preclinically verified. Applicants should state how the proposed research will impact patients with neuromuscular diseases. Applicants are highly encouraged to engage clinicians and/or patients from the Stanford SMA clinic for input on proposals and throughout the duration of the grant. Please describe how you will incorporate this input in your proposal.
    • References – You may include citations for up to five relevant publications.
  3. NIH-format biosketch for each investigator

Applications should be submitted to IMA via the ChEM-H SlideRoom portal. You do not need to submit your applications to your Research Process Manager (RPM) in RMG or through your Office of Sponsored Research (OSR) Contract and Grant officer (CGO) for their approval at this time.

Selection Process: Proposals will be reviewed by a faculty panel knowledgeable in translational research and evaluated based on the strength and novelty of the therapeutic hypothesis and the proposed research plan. Preference will be given to projects that incorporate collaborations consisting of interdisciplinary teams across the university and that incorporate input from clinicians and patients. Final selections will be contingent on shortlisted faculty presenting their proposals to the SMA Foundation.

Questions and Contact Info: Amy Jacobson, PhD, Scientific Program Manager, amy.jacobson@stanford.edu.