hfCas12Max

hfCas12Max is a next-generation, high-fidelity CRISPR-Cas12 nuclease engineered for precise genome editing. Developed by HuidaGene Therapeutics using their proprietary HG-PRECISE platform, it features superior on-target editing efficiency and reduced off-target activity compared to earlier nucleases. Its compact size (1,081 amino acids) allows efficient packaging into single viral vectors such as adeno-associated virus (AAV), which is critical for therapeutic delivery in cell and gene therapy applications. The enzyme recognizes 5′-TTN-3′ PAM sequences and has demonstrated robust performance in mammalian cells and plants. hfCas12Max is being used as the core genome-editing component in clinical-stage therapies such as HG302 for Duchenne muscular dystrophy (DMD), where it enables single-cut exon skipping to restore dystrophin expression with minimal off-target effects. It is also being explored for other indications including neuromuscular disorders, amyotrophic lateral sclerosis (ALS), and neovascular age-related macular degeneration (AMD). The system can be delivered via AAV vectors together with guide RNAs targeting specific genomic loci[1][2][3][4][5][6][7][8].

Other names

high-fidelity Cas12 nucleasehigh-fidelity Cas12i variant