A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
Investigators at University of British Columbia have reported the precise cellular populations responsible for the inability to regenerate muscle tissues in muscular dystrophy.
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.
