HuidaGene Announces Broader Adoption of hfCas12Max Through Sublicensing Agreement to Advance Clinical Cell Therapy Programs

SHANGHAI and MIDDLETOWN (DE), April 9, 2025 — HuidaGene Therapeutics ("HuidaGene"), a global clinical-stage biotechnology company advancing next-generation genomic medicines, today announced the expansion of its high-fidelity CRISPR-Cas12 platform, hfCas12Max, into new therapeutic areas through sublicensing by Synthego Corporation ("Synthego") to Vita Therapeutics, Inc. ("Vita").

Under the sublicense agreement, Vita gains access to HuidaGene's engineered hfCas12Max nuclease and optimized guide RNAs for use in preclinical and clinical applications, including the development of hypoimmunogenic iPSC-based cell therapies targeting neuromuscular disorders.

This sublicensing milestone underscores the significant value and broad applicability of HuidaGene's proprietary hfCas12Max platform. Initially licensed to Synthego in 2024 for manufacturing, commercialization, and sublicensing, hfCas12Max continues to gain industry recognition for its superior on-target editing efficiency, reduced off-target effects, and compact size, ideal for therapeutic delivery.

"The expansion of hfCas12Max into clinical cell therapy programs underscores the growing influence of HuidaGene's genome editing innovations across various therapeutic areas," stated Alvin Luk, Ph.D., M.B.A., Chief Executive Officer and Co-founder of HuidaGene. "We are proud to witness our high-fidelity CRISPR technologies facilitating pioneering treatments beyond our own pipeline, reinforcing our mission to deliver transformative, one-time cures to patients worldwide."

hfCas12Max was developed through HuidaGene's proprietary AI-powered, CRISPR-based HG-PRECISE^Ò platform, a leading technology engine for engineering novel DNA editors, RNA editors, and epigenetic regulators. Its broader adoption through partnerships further strengthens HuidaGene's leadership position in the field of genome editing.

For more information about the strategic licensing agreement for hfCas12Max CRISPR nuclease between Synthego and Vita, please visit www.synthego.com. 

About HuidaGene

HuidaGene utilizes its proprietary AI-driven, CRISPR-based HG-PRECISE^Ò platform to develop potentially curative genome medicine. The company's current clinical programs include:

• HG004gene replacement therapy (ODD & RPDD from the US FDA and ODD from EMA) for RPE65-associated retinal disease (IRD)

• 'LIGHT' first-in-human trial (NCT06088992)

• China's first 'STAR' Phase 1/2 multi-national, "master protocol" clinical trial (NCT05906953)

• HG202 CRISPR/RNA-editing therapy for neovascular age-related macular degeneration (AMD)

• 'SIGHT-I' first-in-human trial (NCT06031727)  

• World's first CRISPR/RNA editing therapy 'BRIGHT' Phase 1 clinical trial (NCT06623279)

• HG204 RNA-editing therapy (ODD & RPDD from FDA and ODD by EMA) for MECP2 duplication syndrome

• World's first CRISPR/RNA-editing 'HERO' for neurodevelopment disorders first-in-human trial (NCT06615206)

• HG302 DNA-editing therapy (ODD & RPDD from FDA) for Duchenne muscular dystrophy (DMD)

• World's first CRISPR/DNA-editing 'MUSCLE' first-in-human trial (NCT06594094)

The preclinical pipelines are advancing simultaneously in HG303 CRISPR/DNA-editing for ALS and CRISPR/RNA-editing therapy for Alzheimer's and Huntington's Disease. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn.