DOD Duchenne Muscular Dystrophy, Idea Development Award
Funding Agency:
- Department of Defense
To meet the intent of the funding opportunity, applications must address opportunities and challenges in the development of safe and effective 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 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 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 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 small animal models that better recapitulate the human pathophysiology to study effectiveness of new therapeutics
• 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.,
Various. The FY24 DMDRP IDA promotes new ideas that are still in the early stages of development and have the potential to yield impactful data and new avenues of investigation. This award supports conceptually innovative, high-risk/high-reward research that could lead to critical discoveries or major advancements that will accelerate progress in improving outcomes for individuals with DMD.
$ 4,480,000
5:00 p.m. Eastern time (ET), August 1, 2024
11:59 p.m. ET, August 22, 2024
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