HUIDAGENE ANNOUNCES IND APPROVAL OF THE FIRST CHINA MULTI-REGIONAL, MULTI-NATIONAL MASTER PROTOCOL OF HG004 BY NMPA

SHANGHAI and CLINTON, N.J., April 18, 2023 /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 China National Medical Products Administration (NMPA) has approved its investigational new drug (IND) application for the planned multi-regional, multi-national clinical trial of HG004 for the treatment of patients suffering from RPE65-associated inherited retinal dystrophies, a group of genetic diseases caused by the mutations in RPE65 gene affecting the retina and passed on to the children. Previously, the company announced HG004 has granted orphan drug designation in Mar-2023 (https://www.prnewswire.com/news-releases/huidagene-receives-orphan-drug-designation-for-gene-therapy-of-blindness-301787815.html) and its multi-national IND has also been cleared in Jan-2023 by the US FDA (https://www.prnewswire.com/news-releases/huidagene-therapeutics-announces-ind-active-for-the-multinational-trial-of-hg004-to-treat-inherited-blindness-301732098.html).

"We are pleased that the China CDE has also granted permission to proceed with this first multi-regional, multi-center gene therapy master protocol in China, which is an important milestone to the company," said Xuan Yao, Ph.D., Co-founder and Chief Executive Officer of HuidaGene. "The company has only founded in 4 years, and we are thrilled to receive the IND clearance from both China and US. I'd like to thank our team for their continuous efforts and the support from WuXi Advanced Therapies and the regulatory authorities. I look forward to advancing the clinical development of HG004 and continuing the development of more innovative gene therapy products with clinically meaningful benefits to patients and families worldwide as soon as possible."

"The clearance by both the China NMPA and US FDA to initiate the first China multi-regional, multi-national, multi-center ocular gene therapy master protocol of HG004 is illustrative to the innovative approach to drug discovery of its Rpe65 gene knockout murine disease model using the CRISPR genome-editing system and global clinical development of the HuidaGene team. The goal of the HG004 program is to develop a one-time, non-AAV2 gene replacement therapy to restore, treat, and prevent blindness of both children and adults with severe visual impairment or blindness due to RPE65-associated retinopathies globally," stated Alvin Luk, Ph.D., Scientific Advisory Board Member of HuidaGene. "The team will deeply and comprehensively investigate the safety, tolerability, efficacy, and clinical durability of HG004 in global clinical development, with the aim of bringing novel modalities with safer and better clinical outcomes to patients."

"Investigational HG004 is a novel ophthalmic injection that is being developed to treat RPE65 retinopathies. Our preclinical data supported our multi-national trial with a starting effective dose (about 25-fold lower vector doses) far below the approved AAV2-hRPE65 gene therapy product with less volume need to be injected into the retina to reduce the risk of AAV vector-associated immunogenicity or ocular adverse events in humans. We already saw a substantial restoration of vision from our adult and pediatric patients dosed with HG004 in our investigator-initiated trial (IIT) at Xinhua Hospital in Shanghai, China," said Dr. Xuan Yao.

About Multi-Regional, Multi-National Clinical Trial of HG004 
HG004 will be evaluated in a multiregional, multinational, multicenter, multiple-cohort, dose-finding study of adult and pediatric subjects with RPE65 retinopathies under one master protocol in different countries. The purposes of the study are to evaluate the safety, tolerability, efficacy, and long-term clinical durability of a single injection of HG004 for up to 52 weeks. Primary endpoints include adverse events, certain laboratory measures, and ophthalmic examinations. The study will also assess visual function via a multiluminance mobility test (MLMT), where subjects will navigate a mobility course under various light levels. After completing the primary study period, subjects will continue to be assessed in a long-term follow-up study of HG004.

About RPE65 Mutation-Associated Inherited Retinal Dystrophies 
Inherited retinal dystrophies (IRDs) are a group of rare blinding conditions caused by mutations in any 1 of more than 250 genes. Leber's congenital amaurosis (LCA), severe early childhood-onset retinal dystrophy (SECORD), early-onset severe retinal dystrophy (EOSRD), and retinitis pigmentosa (RP), which may all be grouped under the heading of RPE65 mutation-associated inherited retinal dystrophies, are considered to represent a phenotypic continuum of the same disease. The RPE65 mutation-associated inherited retinal dystrophies with a typical onset between birth and five years of age exhibit several common clinical findings, chiefly night blindness (light staring with profound nyctalopia and nystagmus), progressive loss of visual fields, and loss of central vision.  The percentage of patients (with biallelic RPE65 mutations) meeting the World Health Organization (WHO) criteria for blindness increased with age and reached 100% after the age of 40 years. Given the often severe and early visual loss associated with RPE65 inherited retinal dystrophies, other areas of development, including speech, social skills, and behavior, may also be delayed.

About HuidaGene -辉大基因 
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine. Based in Shanghai and New Jersey, HuidaGene is committed to addressing patients' needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology. We are currently advancing clinical programs in RPE65 mutation-associated 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. Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. HuidaGene is committed to transforming the future of genome-editing medicine.

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