02.11.2023 – 01:59
Huidagene Therapeutics
Shanghai and Clinton, NJ (ots/PRNewswire)
HG004 is the first AAV gene therapy developed in China to be studied in a multinational master protocol clinical trial for patients with inherited retinal dystrophies caused by mutations in the RPE65 gene. HG004 is also the first gene therapy independently developed in China to be tested by the U.S. FDA has received both ODD and RPDD.HG004 is the Company’s first product candidate from a robust pipeline of advances in ophthalmology and neurology to reach the clinic
Huidagene Therapeutics (辉大基因，“HuidaGene”), a global clinical-stage biotechnology company focused on the development of CRISPR-based programmable genomic medicines, today announced that the first patient in the multinational master protocol clinical trial the phase 1/2a (NCT05906953; CTR20232920) of the gene therapy drug HG004 or the STAR study (Safety and efficacy Trial of HG004 for Leber congenital Amaurosis related to Rpe65 gene mutations), an adeno-associated virus 9 (AAV9) gene replacement therapy candidate for treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRD).
“The common use of AAV2 for RPE65-related retinal diseases requires a high total vector dose and large injection volume, which leads to safety concerns such as retinal detachment, etc.,” said Alvin Luk, Ph.D., MBA, CCRA, co-founder and chief executive officer of HuidaGene “The efficient retinal transduction in the targeted RPE layers using HG004 for the treatment of retinal diseases, allowing to reduce both the total vector dose and the injection volume, was validated in the clinical trial HG00401 for the treatment of adults and children with LCA2 and makes it likely that this therapy can also treat IRD caused by the RPE65 gene, as proposed by the US FDA. There is a huge unmet need for the over 55,000 patients living with RPE65-IRD worldwide. All at HuidaGene is pleased with this significant milestone as we work closely with our sites in China and the US to develop a safer and more effective “one-shot” therapy for the RPE65-IRD patient community worldwide.”
HG004 is a gene replacement therapy product independently developed by HuidaGene. HG004 therapy uses the recombinant AAV9 vector (AAV9) to deliver a functional human RPE65 gene into the retina to restore, treat and prevent blindness in children and adults with RPE65-IRD. HG004 received both Orphan Drug Designation ( and Pediatric Disease Designation ( ) from the US Food and Drug Administration (FDA) this year. The clinical trial HG00401 (NCT06088992) for Leber congenital amaurosis type 2 (LCA2), in which The same product used as HG004 in this multinational, multicenter, master protocol clinical trial (HG00402; NCT05906953) for RPE65-IRDs, also recently announced that the final patient dose resulted in significant improvement in vision and no safety concerns in both adults as well as with children.
“Following the completion of the last patient dose in the IIT study on October 24, the successful completion of the first patient dose in the multinational HG004 study this week means the company has reached another important milestone, particularly in terms of global presence. “We will work diligently to advance this global multinational Phase 1/2a master protocol clinical trial to support concurrent approval and commercialization of the drug in the future,” said Dr. Xuan Yao, co-founder, president and GM for the Greater China from HuidaGene. “This milestone underscores our commitment to developing novel genetic medicines for patients with high unmet needs and our team’s strong execution capabilities. We hope that this multi-regional study, which has the potential to be approved worldwide, will generate new results “May bring transformative therapeutic options to people living with RPE65-IRD.”
About RPE65 Mutation-Associated Inherited Retinal Dystrophies (RPE65-IRD) Inherited retinal dystrophies (IRDs) are a group of rare blinding diseases caused by mutations in one of more than 250 genes. Leber congenital amaurosis (LCA), severe neonatal retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP), all under the heading of IRDs caused by mutations in the RPE65 gene (RPE65-IRDs ) can be summarized and are considered as a phenotypic continuum of the same disease. The RPE65-IRDs, which typically occur between birth and five years of age, share several common clinical findings, most notably night blindness (light staring with marked nyctalopia and nystagmus), progressive visual field loss, and loss of central vision. Given the often severe and premature vision loss associated with RPE65-IRDs, other areas of development such as language, social skills, and behavior may also be delayed.
Information about the multinational clinical trial of HG004 NCT05906953; CTR20232920) This multinational, multi-center, multi-cohort, dose-finding, master protocol clinical trial is designed to evaluate the safety, tolerability and efficacy of HG004 in patients with inherited retinal dystrophies due to RPE65 mutations. Primary endpoints include adverse events, certain laboratory values and ophthalmological examinations. This HG00402 study also examines visual function. Multi-luminance mobility test (MLMT), in which the test subjects complete a mobility course in different lighting conditions. After completion of the primary study period, subjects will continue to be studied in a long-term follow-up study with HG004. For more information, please contact: [email protected] or visit http://clinicaltrials.gov
Informationen zu HuidaGene – Huida Gene
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focused on the discovery, engineering and development of novel gene editing tools and gene therapies to reshape the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene aims to meet the needs of patients worldwide with various preclinical therapeutic programs in the areas of ophthalmology and neurology. We are currently advancing clinical programs for HG004 for the treatment of inherited retinal diseases caused by RPE65 mutations. This program has received both ODD and RPDD from the US Food and Drug Administration (FDA). We are also working on HG202, a CRISPR/Cas13Y RNA editing for neovascular age-related macular degeneration (nAMD), as well as on our preclinical pipeline. This includes programs such as HG301, a CRISPR/Cas12 DNA editing for retinitis pigmentosa, HG204, a CRISPR/Cas13Y RNA editing for the neurodevelopmental disease MECP2 duplication syndrome (MDS) – for which both the RPDD and the ODD are approved by the US FDA were awarded – and HG302, a CRISPR/Cas12 DNA editing for neuromuscular diseases such as Duchenne muscular dystrophy (DMD) and more. The Company’s CRISPR-based therapeutics offer the potential to cure patients with life-threatening diseases by addressing the cause of their disease. HuidaGene is committed to changing the future of genome editing medicine.
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