China-based biotechnology company HuidaGene Therapeutics has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its CRISPR-Cas12 DNA editing therapy, HG302, intended for the treatment of Duchenne muscular dystrophy (DMD), a severe and often fatal neuromuscular genetic disorder. This designation follows the Rare Pediatric Disease Designation (RPDD) that HG302 was granted by the FDA just last month.
HG302 is engineered to deliver high-fidelity Cas12 (hfCas12Max) and CRISPR RNA to the splice donor (SD) site of exon 51 in the human DMD gene, directly to the muscles via a single viral vector. By disrupting this specific site, HG302 induces a skip of exon 51, which results in the formation of a correct open reading frame from exons 50 to 53. This restoration of the reading frame is intended to lead to the expression of functional muscular dystrophy proteins, thereby improving muscle function in children affected by DMD.- Flcube.com
