# Optimization of an integrin enhancing molecule for the treatment of Duchenne muscular dystrophy

> **NIH NIH R44** · STRYKAGEN CORPORATION · 2024 · $994,272

## Abstract

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 α7β1
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 α7 integrin
alleviates disease progression and improves survival of mouse models of DMD. Loss of the α7 integrin in
dystrophin-deficient mdx mice results in severe muscle disease. Together these studies demonstrate that the
α7β1 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 α7 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 α7β1 integrin enhancing molecules, towards IND and clinical trials
for patients with DMD.

## Key facts

- **NIH application ID:** 10930004
- **Project number:** 5R44OD030543-05
- **Recipient organization:** STRYKAGEN CORPORATION
- **Principal Investigator:** Ryan David Wuebbles
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $994,272
- **Award type:** 5
- **Project period:** 2015-04-01 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10930004

## Citation

> US National Institutes of Health, RePORTER application 10930004, Optimization of an integrin enhancing molecule for the treatment of Duchenne muscular dystrophy (5R44OD030543-05). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10930004. Licensed CC0.

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