Asha Therapeutics, Tampa, FL
Testing novel SARM1 inhibitor drug for CMT2A
Asha Therapeutics is looking to test the therapeutic activity of their novel SARM1 inhibitor as a potential treatment option for CMT2A.
CMT2A is caused by mutations in the MFN2 gene, leading to mitochondrial dysfunction and nerve degeneration. Prior studies have shown that genetic removal of SARM1, a key driver in nerve degeneration, prevents disease in CMT2A animals. These findings suggest that pharmacological inhibition of SARM1 could offer a promising therapeutic strategy for CMT2A. ASHA-624 is a novel SARM1 inhibitor developed by Asha Therapeutics that acts as an intramolecular glue, selectively blocking SARM1 activation.
This project will test the ability of ASHA-624 to correct CMT2A disease symptoms in cell and animal models of CMT2A.
We deeply value the support of CMT Research Foundation in evaluating our asset’s novel method of SARM1 inactivation in CMT2A. Asha’s research partnership with CMT Research Foundation will advance critical understandings into ASHA-624’s efficacy and safety for patients with CMT2A, expanding the patient impact potential of SARM1 inactivation across a growing number of neurodegenerative and rare diseases towards the design of disease modifying and curative medicines.