HuidaGene to Highlight Latest Advances in Retina Portfolio at the European Society of Retina Specialists (EURETINA) 2024 Annual Meeting

SHANGHAI and CLINTON (NJ), September 2, 2024 - HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company developing potentially curative genomic medicines, today announced that five presentations will be featured during the European Society of Retina Specialists (EURETINA) 2024 annual meeting, which will take place in Barcelona, Spain, from September 19 to 22.  The Company’s presentations will include the safety and efficacy of the first-in-human clinical trial of HG004 (AAV9-hRPE65) to treat inherited retinal disease caused by RPE65 mutations (LB24-107-9143) and Leber’s congenital amaurosis (CA24-2202-9143) as well as the nonclinical study of HG004 to support clinical trial (CA24-2421-9975).  In addition, the Company will also present the efficacy and safety of the world’s first CRISPR-Cas13 RNA-targeting therapy of HG202 (AAV-Cas13-gRNA-VEGFA) for neovascular age-related macular degenerations (AMD) in mice and non-human primates (CA24-2491-9975) and the clinical study in anti-VEGF treatment-resistant AMD patients (CA24-2505-9143).

“These data at EURETINA 2024 bring us one step closer to a new era of innovation in the retina. As seen in the recent real-world post-marketing studies of LUXTURNA^®(AAV2-RPE65 gene therapy), at least 30% of patients injected with LUXTURNA experienced retinal detachment, macular holes, or progressive chorioretinal atrophy as also seen in many AAV2-RPE65 gene therapy trials in the US and China,” said Alvin Luk, Ph.D., MBA, CCRA, Co-Founder and Chief Executive Officer of HuidaGene. “Our ‘LIGHT-I’ first-in-human clinical study (NCT06088992) of HG004 for inherited blindness shows a superior safety profile and visual improvement than the approved LUXTURNA. Additionally, our ‘SIGHT-I’ study (NCT06031727) of HG202 CRISPR RNA-targeting therapy for AMD demonstrates that one injection of our novel Cas13 packaged into a single AAV vector to knock down the mRNA of VEGFA partially can treat AMD patients who are resistant to anti-VEGF therapies. We’re committed to building on our novel gene therapy and gene editing platform with boldness and patient safety as the highest priority to restore and preserve vision for those living with retinal diseases.”

“Our HG004 strategy of using a better AAV viral vector of RPE tropism aims to reduce the total vector doses and the injection volume; therefore, we can minimize the AAV-related immune response and decrease other safety risks as seen in other AAV2-RPE65 gene therapies,” said Linyu Shi, Ph.D., Co-Founder and Chief Scientific Officer of HuidaGene.  “Up to 46% of AMD patients using anti-VEGF reagents have shown poor response or have developed tachyphylaxis with anti-VEGF therapies. Furthermore, long-term overexpressing of VEGF antibodies may excessively inhibit VEGF's function and affect angiogenesis's function. Hard-to-treat or treatment-resistant anti-VEGF therapies have a devastating impact on AMD patients and their quality of life, suggesting a huge unmet need. Therefore, my team and I developed a CRISPR-Cas13 RNA-target gene editing strategy to suppress VEGFA expression.”

Below is a complete listing of the Company’s abstract featured at the EURETINA Annual Meeting. Abstracts can also be found on the EURETINA website (https://euretina.org/barcelona-2024/abstracts/).

About HuidaGene

HuidaGene utilizes its proprietary CRISPR-based HG-PRECISE^® platform to discover, engineer, and develop genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted ODD and RPDD by FDA), which is currently in the first-in-human ‘LIGHT’ trial (NCT06088992) and Phase 1/2 international, material-protocol ‘STAR’ clinical trial (NCT05906953) as well as CRISPR RNA-editing therapy of HG202 in neovascular age-related macular degeneration first-in-human ‘SIGHT-I’ trial (NCT06031727), and the preclinical pipeline, including HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted ODD and RPDD by FDA and ODD by EMA), HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted ODD and RPDD by FDA), HG303 CRISPR DNA-editing for Amyotrophic Lateral Sclerosis, and CRISPR RNA-editing therapy for Alzheimer’s disease. HuidaGene’s extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn.