Duchenne Muscular Dystrophy
NEWS RELEASE
Released: April 5, 2024
Department of Defense
Congressionally Directed Medical Research Programs (CDMRP)
Duchenne Muscular Dystrophy Research Program (DMDRP)
Anticipated Funding Opportunities for Fiscal Year 2024 (FY24)
The FY24 Defense Appropriations Act provides funding for the Duchenne Muscular Dystrophy Research Program (DMDRP) to support research addressing Duchenne Muscular Dystrophy (DMD) pathobiology, diagnosis, and treatment. The managing agent for the anticipated program announcements/funding opportunities is the CDMRP at the U.S. Army Medical Research and Development Command (USAMRDC).
The DMDRP is providing the information in this pre-announcement to allow investigators time to plan and develop ideas for submission to the anticipated FY24 funding opportunities. This pre-announcement should not be construed as an obligation by the government. The FY24 DMDRP funding opportunity announcements for the following award mechanisms will be posted on the Grants.gov website. Pre-application and application deadlines will be available when the announcements are released.
All applications for the FY24 DMDRP Idea Development Award must address opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that focus on the primary pathology of DMD. Therapies that will be efficacious across the life span, including infants, toddlers, and non-ambulatory individuals, are strongly encouraged.
Studies proposed under this award may include:
- Preclinical testing of combination therapies with small molecules and/or biologics that have existing human clinical data in DMD or are repurposed from other disorders
- Optimized delivery to specific tissues or cell types, including targeting to skeletal muscle, heart, brain, and muscle stem cells (e.g., ligand-assisted delivery, tissue-specific promoters, nanoparticles, alternative vectors, identification of biological barriers to delivery)
- Strategies to overcome preexisting immunity and to facilitate repeat administration of biologic therapies (e.g., immune system modulation, vector modification)
- Cell-based therapies, including, but not limited to, selection of novel cell types, expansion, cell delivery and homing, differentiation, and integration
- Non-genomic therapies that are downstream from the genetic defect to address disease pathogenesis (e.g., mitochondrial dysfunction, inflammation, fibrosis, fatty infiltration, etc.)
- Research that will inform and improve therapy in individuals ineligible for currently approved or in-development therapeutics (e.g., age, type of mutations, seropositivity, rare exons)
- Discovery and validation of novel targets, including genetic modifiers and factors that determine the selective vulnerability/resistance of individual muscles, especially in humans
- Validation of novel models that better recapitulate the human pathophysiology
- Therapies that address muscle regeneration deficits
Studies in the following research areas will not be supported by this award mechanism:
- Studies testing the efficacy of candidate therapeutics without a strong mechanistic rationale
- Evaluation of vectors or delivery technologies already prevalent in research studies (e.g., AAV9)
All applications for the FY24 DMDRP Clinical/Translational Research Award must address at least one of the following Focus Areas:
Pre-Clinical Translational Research
- Extension or expansion of existing preclinical data in support of Investigational New Drug application-enabling studies. For example:
- Optimizing delivery to target tissues
- Drug exposure
- Independent replication
- Comparative studies
- Assay development, outcome measures, and/or biomarkers (e.g., pharmacodynamic, prognostic, and predictive biomarkers, including potential surrogate markers)
Clinical Research
- Clinical studies designed to improve care and quality of life
- Prospective real-world data collection for combination or sequential therapies, and/or long-term safety and efficacy studies
- Assessment of clinical trial tools and outcome measures
- In understudied systems (e.g., cognitive, cardiac, and/or gastrointestinal [GI]) or age ranges (e.g., infants, toddlers, and/or non-ambulatory).
- Discovery and qualification of pharmacodynamic, prognostic, and predictive biomarkers, including potential surrogate markers
- Novel clinical outcome assessment
- Patient-centered outcomes (e.g., quality of life, activities of daily living)
- Secondary data analysis that helps to address clinical research tool validation
- Natural history studies in understudied systems (e.g., cognitive, cardiac, and/or GI) or age ranges (e.g., infants, toddlers, and/or non-ambulatory) with an aim toward clinical trial readiness
Award Mechanism | Eligibility | Key Mechanism Elements | Funding |
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Idea Development Award | Established Investigators: Independent investigators at or above the level of Assistant Professor (or equivalent).
New Investigators - Early Stage: Investigators that meet the following criteria by the application submission deadline date:
New Investigators - Transitioning: Established investigators in an area other than muscular dystrophy at or above the level of Assistant Professor seeking to transition to a career in DMD, thereby bringing their expertise to the field. Investigators must pursue an active line of research in DMD and commit at least 10% of their efforts during each budget year toward the proposed DMD research project. |
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Clinical/Translational Research Award | Independent investigators at all academic levels Early-Career Partnering Principal Investigator (PI) Option:
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The following are general descriptions, although not all-inclusive, of the scope of research projects that would be appropriate to propose under each funding level:
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Research Level 1
Research Level 2
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A pre-application is required and must be submitted through the electronic Biomedical Research Application Portal (eBRAP) at https://ebrap.org/ prior to the pre-application deadline. All applications must conform to the final funding opportunity announcements that will be available for downloading from the Grants.gov website. The application package containing the required forms for each award mechanism will also be found on Grants.gov. A listing of all CDMRP and other USAMRDC extramural funding opportunities can be obtained on the Grants.gov website by performing a basic search using CFDA Number 12.420.
Submission deadlines are not available until the funding opportunity announcements are released. For email notification when announcements are released, subscribe to program-specific news and updates under "Email Subscriptions" on the eBRAP homepage at https://ebrap.org/. For more information about the DMDRP or other CDMRP-administered programs, please visit the CDMRP website (https://cdmrp.health.mil).
Point of Contact:
CDMRP Public Affairs
301-619-9783
usarmy.detrick.medcom-cdmrp.mbx.cdmrp-public-affairs@health.mil
Last updated Friday, April 5, 2024