HuidaGene Therapeutics, based in China, has announced that it has received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for its CRISPR-Cas12 DNA editing therapy, HG302. This designation highlights the therapy’s potential to treat Duchenne muscular dystrophy (DMD), a severe neuromuscular genetic disorder.
HG302 employs high fidelity Cas12 (hfCas12Max) combined with CRISPR RNA to target the splice donor (SD) site of exon 51 within the human DMD gene. The therapy is delivered to muscle tissues via a single viral vector, effectively disrupting the targeted site. This disruption enables the skipping of exon 51, thereby creating a corrected open reading frame from exons 50 to 53. The outcome is the restoration of functional dystrophin proteins, which is expected to enhance muscle function in children afflicted with DMD.- Flcube.com
