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.
The CRISPR genome editing technique has been used to activate a close relative of the gene that is mutated in muscular dystrophy type 1A, preventing development of the disease in mouse-model neonates and reversing symptoms in mice with established pathology.