Duchenne Muscular Dystrophy
Released: May 5, 2022
Department of Defense
Congressionally Directed Medical Research Programs (CDMRP)
Duchenne Muscular Dystrophy Research Program (DMDRP)
Anticipated Funding Opportunities for Fiscal Year 2022 (FY22)
The FY22 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 FY22 funding opportunities. This pre-announcement should not be construed as an obligation by the government. The FY22 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.
The Programmatic Panel members recommended that all applications for the for the FY22 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, particularly in adolescents and adults, are encouraged.
Studies proposed under this award may include:
- Optimized delivery to affected tissues, including specific targeting to skeletal muscle, heart, and brain (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)
- Therapeutics targeted to muscle stem cells
- Cell-based therapies, including but not limited to, selection of novel cell types, expansion, cell delivery and homing, differentiation, and integration
- 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
- Therapies addressing secondary pathologies of DMD to the extent that they augment therapies directed at primary disease mechanisms
- 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
- Small molecule studies in animals and humans, either individually or in combination with other molecules and/or biologics
Studies in the following research areas will not be supported by this award mechanism:
- Studies testing the efficacy of candidate therapeutics that are not fully characterized or lack a mechanistic rationale
- Evaluation of vectors or delivery technologies already prevalent in research studies (e.g., AAV9)
The Programmatic Panel members recommended that all applications for the FY22 DMDRP Translational Research Award must address at least one of the following Focus Areas:
- Translational and clinical studies, novel interventions, and drug and biologic delivery technologies designed to improve care and quality of life in areas such as:
- Cognitive function
- Endocrinology (including bone health and growth)
- Skeletal muscle
- Assessment of clinical trial tools and outcome measures such as:
- 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 and/or to understand natural history
- Extension or expansion of existing preclinical translational data in support of a specific therapeutic development path (such as optimizing delivery to target tissues, including drug exposure, independent replication, and comparative studies)
|Award Mechanism||Eligibility||Key Mechanism Elements||Funding|
|Idea Development Award
Modified for FY22
|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
Modified for FY22
|Investigators at or above the level of Assistant Professor (or equivalent)
Optional Early-Career Partnering PI:
Early-Career Partnering PI Option:
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.army.mil).
Point of Contact:
CDMRP Public Affairs
Last updated Thursday, May 5, 2022