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Duchenne Muscular Dystrophy

NEWS RELEASE

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:

  • Have not previously received a DMDRP Idea Development Award.
  • Are within 10 years of first faculty appointment (or equivalent).

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.
  • Supports the development of innovative, high-impact ideas that advance the understanding of DMD and ultimately lead to improved outcomes.
  • Must address opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that address primary pathology of DMD. Therapies that will be efficacious across the lifespan, including infants, toddlers, and non-ambulatory individuals, are strongly encouraged.
  • Preliminary data required.
  • Clinical trials or applications including a clinical trial aim are not allowed.
  • Maximum funding of $350,000 in direct costs (plus indirect costs).
  • Maximum period of performance is 2 years.
Translational Research Award Independent investigators at all academic levels

Early-Career Partnering Principal Investigator (PI) Option:
  • The Partnering PI must be an independent, early-career investigator within 10 years of their first faculty appointment by the time of application submission. Lapses in research time or appointments as denoted in the biographical sketch should be explained in the application.
  • It is encouraged, but not required, that the partnering PI is a clinician or clinical scientist (M.D. or M.D./Ph.D.) to increase collaboration between clinical and non-clinical aspects of DMD research.
  • Supports advanced translational studies that have moved beyond the realm of basic research and have the potential to result in a near-term impact in clinical research or the clinic.
  • Research projects investigating therapies that will be efficacious across the lifespan, including infants, toddlers, and non-ambulatory individuals, are strongly encouraged.
  • Preliminary data required.
  • Clinical trials are allowed.
  • Maximum funding of $1,250,000 in direct costs (plus indirect costs).
  • Maximum period of performance is 3 years.

Early-Career Partnering PI Option:

  • Maximum funding of $1,350,000 in direct costs (plus indirect costs).
  • Maximum period of performance is 3 years.

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
301-619-9783
usarmy.detrick.medcom-cdmrp.mbx.cdmrp-public-affairs@health.mil


Last updated Friday, May 5, 2023