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HuidaGene Therapeutics’ CRISPR Therapy Earns Orphan Drug Status for Duchenne Muscular Dystrophy
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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…
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HuidaGene Secures Rare Pediatric Disease Designation for CRISPR Therapy HG302 Targeting DMD
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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)…
