HG302

HG302 is a first-in-class, CRISPR-based DNA-editing therapy developed by HuidaGene Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). It uses HuidaGene’s proprietary high-fidelity Cas12i variant (hfCas12Max) and a guide RNA (gRNA) to target the exon 51 splice-donor site of the human DMD gene, inducing exon 51 skipping. This edit restores the open reading frame, enabling production of a truncated but functional dystrophin protein and improving muscle function. Unlike previous therapies relying on high-dose AAV vectors or micro-dystrophin gene replacement, HG302 delivers its genome editing machinery in a single AAV vector at lower doses, minimizing risk of immune-related adverse effects. HG302 has shown promising preclinical results (restored dystrophin, improved muscle function) and is being investigated as a one-time intravenous treatment, with the MUSCLE phase 1/2 clinical trial currently underway[1][2][3][4][5][7].