Scienza e Tecnologia
HUIDAGENE THERAPEUTICS ANNOUNCES FIRST PATIENT DOSED IN MULTNATIONAL PHASE 1/2 TRIAL OF HG004 FOR INHERITED BLINDNESS
HG004 is an investigational gene replacement therapy product independently-developed by HuidaGene. This HG004 therapy uses the recombinant AAV9 (AAV9) vector to deliver a functional human gene to the retina and to restore, treat, and prevent blindness of children and adults with -IRDs. HG004 was granted both orphan drug designation (https://www.prnewswire.com/news-releases/huidagene-receives-orphan-drug-designation-for-gene-therapy-of-blindness-301787815.html) and rare pediatric disease designation (https://www.prnewswire.com/news-releases/huidagene-therapeutics-receives-fda-rare-pediatric-disease-designation-for-hg004-to-treat-inherited-blindness-301894514.html) by the U.S. Food and Drug Administration (FDA) this year. The HG00401 clinical trial (NCT06088992) for Leber congenital amaurosis type 2 (LCA2) using the same product of HG004 in this multinational, multi-center, master-protocol clinical trial (HG00402; NCT05906953) for -IRDs also recently announced that the last-patient-dose has been completed with significant visual improvement and no safety concerns in both adult and pediatric populations.
"Following the completion of last-patient-dose in the IIT study on October 24 , the successful completion of first-patient-dose in the HG004 multinational trial this week means that that company has reached another important milestone, particularly for the global footprint. We'll work diligently to advance this global multinational, master-protocol Phase 1/2a clinical trial to support simultaneous drug marketing approval and commercialization in the future," said Xuan Yao , Ph.D., Co-founder, President, and GM for Greater China of HuidaGene. "This milestone highlights our commitment to developing novel genetic medicines to patients with high, unmet needs and the strong execution capabilities of our team. We hope that this multi-regional trial with the potential to be registrational globally can bring new, transformative therapeutic options for individuals living with -IRD worldwide."
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 IRDs caused by the mutations in gene ( -IRDs), are considered to represent a phenotypic continuum of the same disease. The -IRDs with a typical onset between birth and 5 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. Given the often severe and early visual loss associated with -IRDs, other areas of development including speech, social skills, and behavior may also be delayed.
This multinational, multicenter, multiple-cohort, dose-finding, and master-protocol clinical trial is designed to evaluate the safety, tolerability, and efficacy of HG004 in patients with inherited retinal dystrophies caused by
mutations. The primary endpoints include adverse events, certain laboratory measures, and ophthalmic examinations. This HG00402 study will also assess visual function via. multi-luminance mobility test (MLMT) where subjects will be navigating a mobility course under a variety of light levels. Following the completion of the primary study period, subjects will continue to be assessed in a long-term follow-up study of HG004. For more information, please contact: HG00402@huidagene.com or visit http://clinicaltrials.gov
HuidaGene Therapeutics (辉大基因) is a global clinical-stage biotechnology company focusing on discovering, engineering, and developing novel gene-editing tools and gene therapies 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 and neurology. We are currently advancing clinical programs of HG004 in inherited retinal disease caused by mutations (which has been granted both ODD and RPDD by U.S. FDA), HG202 CRISPR/Cas13Y RNA-editing in neovascular age-related macular degeneration (nAMD), and our preclinical pipeline, including programs HG301 CRISPR/Cas12 DNA-editing in retinitis pigmentosa, HG204 CRISPR/Cas13Y RNA-editing in neurodevelopmental disease of
duplication syndrome (MDS) (which has been granted RPDD and ODD by U.S. FDA), and HG302 CRISPR/Cas12 DNA-editing in neuromuscular diseases of Duchenne muscular dystrophy (DMD) etc. 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.
For more information, please visit http://www.huidagene.com
or follow us on LinkedIn at http://www.linkedin.com/company/huidagene
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