Duchenne Muscular Dystrophy
Released: May 5, 2023
Department of Defense
Congressionally Directed Medical Research Programs (CDMRP)
Duchenne Muscular Dystrophy Research Program (DMDRP)
Anticipated Funding Opportunities for Fiscal Year 2023 (FY23)
The FY23 Defense Appropriations Act provides funding for the DMDRP to support research addressing Duchenne Muscular Dystrophy (DMD) pathobiology, diagnosis, and treatment. The managing agent for the anticipated 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 FY23 funding opportunities. This pre-announcement should not be construed as an obligation by the government. The FY23 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 FY23 DMDRP Idea Development Award must address opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that focus on primary pathology of DMD. Therapies that will be efficacious across the life span, including infants, toddlers, and non-ambulatory individuals, are 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 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 older individuals, including dosing challenges, genetic-based therapies, tissue environments, and other factors that may compromise delivery and efficacy in this patient group
- 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 small animal models that better recapitulate the human pathophysiology
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 FY23 DMDRP Translational Research Award must address at least one of the following Focus Areas:
- Investigational New Drug (IND)-enabling or clinical studies designed to improve care and quality of life
- Assessment of clinical trial tools and outcome measures with a focus on understudied systems (e.g., cognitive, cardiac, and/or gastrointestinal [GI]) or age ranges (e.g., infants, toddlers, and/or non-ambulatory). For example:
- 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
- Extension or expansion of existing preclinical data in support of a pre-IND regulatory submission, such as optimizing delivery to target tissues, including drug exposure, independent replication, and comparative studies
- 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|
|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.
|Translational Research Award||Independent investigators at all academic levels
Early-Career Partnering Principal Investigator (PI) Option:
Early-Career Partnering PI Option:
A pre-application is required and must be submitted through the Biomedical Research Application Portal (eBRAP) 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. For more information about the LCRP or other CDMRP-administered programs, please visit the CDMRP website (https://cdmrp.health.mil).
Point of Contact:
CDMRP Public Affairs
Last updated Friday, May 5, 2023