Huidagene Therapeutics
SHANGHAI, March 20 /PRNewswire/
HuidaGene Therapeutics (辉大基因; HuidaGene), a global clinical-stage biotechnology company focused on the development of genomic medicines, announces that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for HG004, HuidaGene’s lead gene replacement therapy for the treatment of inherited retinal diseases caused by RPE65– mutations are caused. Patients who attend with RPE65– Mutations associated with inherited retinal diseases (IRDs), a group of rare blinding diseases caused by biallelic RPE65-mutations suffer from retinal impairments, which are also passed on to the offspring.
“We are pleased about this important confirmation from the US Food and Drug Administration. Receiving orphan drug designation is an important milestone on the path to clinical testing of our HG004 gene replacement therapy program, which will treat children and adults affected by RPE65-mutation-related hereditary retinal diseases, to provide a safe, durable and high-quality treatment,” said Xuan Yao, PhD, Co-Founder and Chief Executive Officer of HuidaGene. “He also emphasizes the importance of bringing this novel therapy to patients with severe visual impairments or blindness, and gives us great motivation to accelerate the clinical development of HG004.”
The Orphan Drug Act encourages the development of treatments, diagnostics, or prevention measures for rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation offers HuidaGene the opportunity to obtain certain benefits, including clinical development tax credits, waivers of certain FDA filing fees, seven years of post-approval market exclusivity, and drug development assistance. Orphan drug status will accelerate drug development and regulatory approval efforts in the United States. HG004 is eligible for certain development incentives, including FDA support for clinical trials. In January 2023, the FDA approved the HG004 application for the planned multinational clinical trial and is currently under review by China’s National Medical Products Administration (NMPA) for this first AAV gene replacement therapy master protocol in various countries including China.
information about with RPE65– Mutations associated inherited retinal diseases Inherited retinal diseases (IRDs) are a group of rare blinding diseases caused by mutations in one of more than 250 genes. Leber’s congenital amaurosis (LCA), severe retinal dystrophy of early childhood (SECORD), early-onset severe retinal dystrophy (EOSRD) and retinitis pigmentosa (RP), all included under the term RPE65-Mutation-associated inherited retinal diseases (IRDs) are considered to be a phenotypic continuum of the same disease. The one with the RPE65mutation-associated inherited retinal disorders, typically occurring between birth and five years of age, share several common clinical findings, most notably night blindness, progressive visual field defects, and loss of central vision. The percentage of patients (with biallelic RPE65mutations) that met World Health Organization (WHO) criteria for blindness increased with age, reaching 100% after the age of 40. Given the often severe and premature vision loss associated with inherited RPE65-retinal diseases, other areas of development such as language and social behavior may also be delayed. In 2023, over 6,600 people in the US are expected to be diagnosed with RPE65-diagnosed with mutation-associated IRD.
Information about HG004 HG004 is a gene replacement therapeutic that uses the recombinant non-adeno-associated virus serotype 2 vector (non-AAV2 vector) to express a functional human RPE65inserting a gene into the retina to prevent blindness in children and adults RPE65-Restore, treat and prevent mutation-associated IRD. The extensive preclinical studies demonstrated superior transduction efficiency of the RPE layer and restoration of retinal functions with HG004 compared to AAV2-mediated gene replacement therapy by the company’s independently developed Rpe65gene knockout (KO) mouse model using the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) genome editing system. The mouse model mimics the retinal phenotypes and functions of patients RPE65-mutation-associated IRDs. Based on the head-to-head preclinical comparison study of HG004 and adeno-associated virus serotype 2 (AAV2) at identical dose, the recovery of retinal functions compared to wild-type mice was im Rpe65knockout mouse model increased by 67.6% (HG004) and by 35.8% (AAV2 products) at 17 weeks after a single injection, suggesting that HG004 may be able to reduce total vector doses to reduce the risk of AAV vector-associated immunogenicity or ocular side effects in humans.
About HuidaGene – 辉大基 因
HuidaGene Therapeutics (辉大基因) is a global clinical development biotechnology company focused on the discovery, design and development of CRISPR-based genetic medicines to transform 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-associated IRD and our preclinical pipeline. These include programs for 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 in medicine.
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