HuidaGene Therapeutics today announced the first patient has been dosed in the multinational, master-protocol Phase 1/2a clinical trial (NCT05906953; CTR20232920) of HG004 gene therapy drug, or the STAR (Safety and efficacy Trial of HG004 for leber congenital Amaurosis related to Rpe65 gene mutations) study, an adeno-associated virus 9 (AAV9) gene replacement therapy candidate for the treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRD).
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[01-November-2023] |
SHANGHAI and CLINTON, N.J., Nov. 1, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因,"HuidaGene”), a global clinical-stage biotechnology company focused on developing CRISPR-based programmable genomic medicines, today announced the first patient has been dosed in the multinational, master-protocol Phase 1/2a clinical trial (NCT05906953; CTR20232920) of HG004 gene therapy drug, or the STAR (Safety and efficacy Trial of HG004 for leber congenital Amaurosis related to Rpe65 gene mutations) study, an adeno-associated virus 9 (AAV9) gene replacement therapy candidate for the treatment of inherited retinal dystrophies caused by RPE65 mutations (RPE65-IRD). “Commonly used AAV2 for RPE65-related retinal diseases requires high total vector dose and large injection volume emerging to safety concerns such as retinal detachment etc,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and Chief Executive Officer of HuidaGene. “The efficient retinal transduction in the targeted RPE layers using HG004 for retinal disease allowing to lower both the total vector dose and injection volume has been validated in the HG00401 clinical trial for treating adults and children with LCA2 and makes it likely that this therapy can also potentially treat the IRD caused by RPE65 gene, as suggested by the U.S. FDA. The unmet need is huge for over 55,000 patients living with RPE65-IRD worldwide. Everyone at HuidaGene is excited about this significant milestone as we’re working closely with the sites in China and U.S. to develop a safer and more efficacious ‘once-and-done’ therapy for the RPE65-IRD patient community globally.” 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 RPE65 gene to the retina and to restore, treat, and prevent blindness of children and adults with RPE65-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 RPE65-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 RPE65-IRD worldwide.” About RPE65 Mutation-Associated Inherited Retinal Dystrophies (RPE65-IRD) About Multinational, Master-Protocol Clinical Trial of HG004 (NCT05906953; CTR20232920) About HuidaGene - 辉大基因 View original content to download multimedia:https://www.prnewswire.com/news-releases/huidagene-therapeutics-announces-first-patient-dosed-in-multinational-phase-12-trial-of-hg004-for-inherited-blindness-301974277.html SOURCE HuidaGene Therapeutics |