Casma Therapeutics Inc. has developed bicyclic heteroaromatic compounds acting as mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of Duchenne muscular dystrophy, neurodegeneration, atherosclerosis, cancer, infections, liver, lysosomal storage and inflammatory bowel diseases.
Casma Therapeutics Inc. has nominated its first development candidate, CSM-101, a first-in-class TRPML1 agonist. CSM-101 is being developed for the treatment of Gaucher’s disease patients with Parkinson’s disease, with the potential to expand into GBA-associated Parkinson’s disease and broader Parkinson’s disease populations.
Casma Therapeutics Inc. has described autophagy targeting chimeras (AUTACs) comprising sequestosome-1 (ubiquitin-binding protein p62; SQSTM1) ligands coupled to an echinoderm microtubule-associated protein-like 4-ALK (EML4- ALK)-targeting moiety via a linker.
In the more than two years since Casma Therapeutics Inc. raised its series A and completed its new $50 million series B, the company has advanced its agonist program for treating muscular dystrophy and identified new targets.
