Optimization of an integrin enhancing molecule for the treatment of duchenne muscular dystrophy
Ended Project Grant Awarded Last action
- Award ID
- R44OD030543
- Recipient
-
Strykagen Corp UEI GZYNUKFEJRN5
- Awarding agency
-
Aggregate Financial Assistance Data
National Institutes of Health, Department of Health and Human Services
- Obligated
- $2,912,218
Award facts
- Assistance listing
- 93.351 Research Infrastructure Programs
- Completion date
- Originally
- Funding agency
- Office of the Director National Institutes of Health, Department of Health and Human Services
- Place of performance
- Reno, NV 89557, USA
Award details come from USAspending.gov.
Description
- Abstract
- Duchenne muscular dystrophy (DMD) is a fatal muscle disease with an incidence of 1 in 5000 males. DMD results from mutations in the gene encoding dystrophin, a 427 KDA scaffolding protein responsible for providing a mechanical link between the muscle fiber actin cytoskeleton and laminin in the extracellular matrix. The A7SS1 integrin is a transmembrane linkage system in skeletal and cardiac muscle that also links laminin to the actin cytoskeleton. Studies have demonstrated that transgenic and virally mediated overexpression of the A7 integrin alleviates disease progression and improves survival of mouse models of DMD. Loss of the A7 integrin in dystrophin-deficient MDX mice results in severe muscle disease. Together these studies demonstrate that the A7SS1 integrin can serve as a surrogate for the loss of dystrophin and is a target for drug-based therapies. The burkin lab has previously shown treatments with compounds that increase the A7 integrin in dystrophic muscle lead to enhanced muscle regeneration, improved skeletal muscle strength and decreased myofiber damage. In the phase 2A sttr, strykagen has shown that treatments with our lead STRYKA969 small molecule are safe, on- target, and have beneficial effects for both dystrophic mouse skeletal and cardiac muscle. In this phase 2B sbir proposal, we propose to complete IND-enabling STRYKA969 tier 2 adme, organ toxicity, and metabolite profiling studies. We will also assess the serum pharmacokinetics of STRYKA969 in both canine and non-human primates. Lastly, we will perform an efficacy study in the large animal grmd DOG model, considered the gold standard animal model for DMD. These studies will scale the therapeutic treatment using STRYKA969 in a large animal preclinical model that more closely recapitulates DMD patient skeletal and cardiac disease. Results from this study will move STRYKA969, as a first in class A7SS1 integrin enhancing molecules, towards IND and clinical trials for patients with DMD.
Transactions
| Action | Obligated | Date |
|---|---|---|
| Amendment | $0 | |
| Amendment | $994,272 | |
| Amendment | $0 | |
| Amendment | $0 | |
| Amendment | $958,973 | |
| Original award | $958,973 |
Quick answers
Who received award R44OD030543?
R44OD030543 was awarded to Strykagen Corp by Aggregate Financial Assistance Data (National Institutes of Health) on April 1, 2015.
How much is award R44OD030543 worth?
$2,912,218 has been obligated on R44OD030543.
Which agency awarded R44OD030543?
Aggregate Financial Assistance Data (National Institutes of Health) awarded R44OD030543, with funding from Office of the Director (National Institutes of Health).
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