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small molecule α7β1 integrin activators
- Development stage
- Preclinical
- Lead developer
- Sarcomatrix Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Sarcomatrix Therapeutics is developing a pipeline of small molecule activators targeting the α7β1 integrin for the treatment of multiple muscular dystrophies, including Duchenne (DMD), Becker (BMD), Emery-Dreifuss (EDMD), Facioscapulohumeral (FSHD), Limb-Girdle (LGMD), and Congenital muscular dystrophy (CMD). α7β1 integrin is a major laminin receptor in skeletal muscle that links the extracellular matrix to the intracellular cytoskeleton. In dystrophin-deficient muscle, such as in DMD and BMD, increasing the expression or activity of α7β1 integrin can compensate for the lack of the dystrophin-glycoprotein complex, thereby stabilizing the muscle cell membrane (sarcolemma) and preventing muscle wasting. These small molecules aim to stimulate muscle regeneration and maintain muscle integrity through a mutation-agnostic approach. The program is currently in the preclinical stage of development.
- Other names
- α7β1 integrin activatorsSarcomatrix α7β1 integrin small moleculessmall molecule α7β1 integrin activators (Sarcomatrix Therapeutics)
02
Targets
α7β1 (Integrin α7β1)
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