Huidagene Therapeutics
SHANGHAI and CLINTON, NJ, April 20 /PRNewswire/
HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage biotechnology company developing CRISPR-based programmable genomic medicine, announces that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has approved its application for an investigational new drug (IND) for the planned multi-regional, multinational clinical trial of HG004 in the treatment of patients with RPE65-associated inherited retinal dystrophies. It is a group of genetic disorders caused by mutations in the RPE65 gene that affect the retina and are passed on to the children. Previously, the company announced that HG004 was granted orphan drug designation in March 2023 ( ) and that the US FDA approved the multinational IND in January 2023 ( ).
“We are pleased that China’s CDE has also granted approval to proceed with this first multi-region, multi-center gene therapy master protocol in China, which is an important milestone for the company,” said Dr. Xuan Yao, co-founder and CEO of HuidaGene. “The company was only founded 4 years ago and we are thrilled to receive IND approval from both China and the US. I would like to thank our team for their continued efforts and the support of WuXi Advanced Therapies and regulators. I I look forward to advancing the clinical development of HG004 and continuing the development of more innovative gene therapy products with clinically meaningful benefits for patients and families worldwide as soon as possible.”
“The approval by both the Chinese NMPA and the US FDA to initiate the first multi-regional, multinational, and multi-center ocular gene therapy master protocol for HG004 in China illustrates the innovative approach to drug discovery for the Rpe65 Gene knockout mouse disease model using CRISPR genome editing system and global clinical development by HuidaGene team. The goal of the HG004 program is to develop a unique, non-AAV2 gene replacement therapy for the vision restoration, treatment and prevention of blindness in children and adults with severe visual impairment or blindness due to RPE65 -associated retinopathies worldwide,” said Dr. Alvin Luk, member of HuidaGene’s Scientific Advisory Board To provide ways with safer and better clinical outcomes.”
“Investigational drug HG004 is a novel eye injection designed to treat RPE65 -Retinopathies is developed. Our preclinical data supported our multinational study with an effective starting dose (approximately 25-fold lower vector doses) well below that of the approved AAV2hRPE65 gene therapy product and where a smaller amount can be injected into the retina to reduce the risk of AAV vector-associated immunogenicity or ocular side effects in humans. “We have already seen substantial vision restoration in our adult and pediatric patients with HG004 in our investigator-initiated study (IIT) at Xinhua Hospital in Shanghai, China,” said Dr. Xuan Yao.
Information on the multi-region, multinational clinical study of HG004
HG004 is being enrolled in a multi-region, multinational, multi-centre, multi-cohort, dose-ranging study in adult and pediatric subjects RPE65 -Retinopathies are examined as part of a master protocol in different countries. The purpose of the study is to evaluate the safety, tolerability, efficacy and long-term clinical durability of a single injection of HG004 over a period of up to 52 weeks. The primary endpoints include adverse events, certain laboratory values and ophthalmological examinations. As part of the study, visual function is also assessed using a multi-luminance mobility test (MLMT), in which the subjects have to complete a mobility course in different lighting conditions. Upon completion of the primary study period, subjects will continue to be evaluated in a long-term follow-up study of HG004.
information about RPE65 Mutation-Associated Inherited Retinal Dystrophies
Inherited retinal dystrophies (IRDs) are a group of rare blindnesses caused by mutations in 1 of more than 250 genes. Leber’s congenital amaurosis (LCA), severe infantile retinal dystrophy (SECORD), early onset severe retinal dystrophy (EOSRD) and retinitis pigmentosa (RP), all under the heading RPE65 Mutation-associated inherited retinal dystrophies are considered to be a phenotypic continuum of the same disease. The one with the RPE65 Mutation-associated inherited retinal dystrophies, which typically appear between birth and five years of age, share several common clinical findings, most notably night blindness (photophobia with marked nyctalopia and nystagmus), progressive visual field defects, and loss of central vision. The percentage of patients (with biallelic RPE65 mutations) that meet World Health Organization (WHO) criteria for blindness increases with age, reaching 100% after age 40. Given the often severe and premature vision loss associated with inherited RPE65 -Retinal dystrophies, other areas of development, including language, social skills, and behavior, may also be delayed.
About HuidaGene – Huida Gene
HuidaGene Therapeutics (辉大基因) is a global, clinical-stage biotechnology company focused on the discovery, engineering and development of CRISPR-based gene medicines to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to serving the needs of patients around the world with various preclinical therapy programs in the fields of ophthalmology, otology, myology and neurology. We are currently driving clinical programs for RPE65 mutation-related inherited retinal dystrophies and our preclinical pipeline, including programs in neovascular age-related macular degeneration, retinitis pigmentosa, hereditary hearing loss, Duchenne muscular dystrophy and MECP2 duplication syndrome. The company’s CRISPR-based therapeutics offer the potential to heal patients with life-threatening diseases by addressing the root cause of their disease. HuidaGene aims to change the future of genome-editing medicine.
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