HuidaGene at CRISPR MEDiCiNE 2025: A Celebration of Progress, Promise, and Patients

SHANGHAI and MIDDLETOWN (DE), April 10, 2025—This week at the CRISPR MEDiCiNE 2025 conference (#CRISPRMED25), the future of CRISPR-based therapies became sharply focused. HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company at the forefront of next-generation genome medicines, was proud to be at the forefront of this transformative movement.

The conference started with exciting news: our high-fidelity nuclease hfCas12Max, sub-licensed to Vita Therapeutics through Synthego, is advancing next-generation iPSC-based therapies globally - a significant step forward for scalable, one-time cures. Throughout the meeting, the scientific energy was palpable. Leaders across academia and industry showcased cutting-edge innovations — from prime and base editing strategies to delivery breakthroughs using engineered capsids to scalable manufacturing for gene-edited therapies.

HuidaGene's Co-founder and CEO, Dr. Alvin Luk, delivered a powerful keynote, unveiling early clinical successes that stirred the audience:

• HG302 (hfCas12Max) for Duchenne muscular dystrophy (DMD) demonstrated strong safety with no SAEs, DLT, or major toxicities, including thrombocytopenia, myocarditis, and thrombotic microangiopathy — and early functional gains in treated boys (+1 North Star Ambulatory Assessment point, +60 meters in 6-minute walk test, and 30 seconds improvement in 4-stair climb just 3 months post-treatment).

• HG204 (hfCas13Y), the world's first RNA-editing therapy for MECP2 duplication syndrome (MDS), showed meaningful cognitive, motor, and emotional improvements in children — a significant breakthrough in tackling a devastating pediatric neurodevelopmental disorder.

The highlight of the week came when heartfelt videos of an MDS boy with an 11-month-old IQ treated with HG204 were shared. These videos showed him walking more steadily, following simple commands such as “come to eat", "go to the bathroom”, and “come to take a bath”, and reconnecting emotionally just 12 weeks after dosing—a vivid reminder that behind every innovation stands a life transformed.

Key conference themes resonated powerfully with HuidaGene's mission:

• Patient access must evolve alongside innovation — requiring global collaboration across regulators, manufacturers, and patient groups.

• Delivery technologies are unlocking new indications, enabling CRISPR therapies to reach more patients with precision and safety.

• Standardization of quality, efficacy, and safety benchmarks will be critical to scaling CRISPR therapies globally.

As we move forward, HuidaGene remains deeply committed to advancing safe, accessible, one-time CRISPR cures, expanding our high-fidelity editing toolbox, and building global partnerships that prioritize patient outcomes.

At CRISPR MEDiCiNE 2025, we didn't just share data — we shared hope. And we are just getting started. The future of CRISPR is here!

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.