HuidaGene Therapeutics Co., Ltd.

SHANGHAI, China and CLINTON, NJ, USA I November 4, 2024 I HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company, announced that the U.S. FDA has cleared its investigational new drug (IND) application for HG202, the world’s first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD), which affects millions worldwide. “This open IND for HG202 by the U.S. FDA-the first regulator to clear CRISPR/Cas13 for clinical development–marks a significant milestone for HuidaGene and the CRISPR RNA-editing field,” noted Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. “The strong preclinical data for HG202, combined with promising results from the first-in-human ‘ SIGHT-I ‘ trial in China, underscored its potential to address AMD using a non-receptor binding pathway through the Cas13 RNA editor to knockdown VEGF-A mRNA.” Xin Zhang, M.D., MSc., COO and CMO of HuidaGene, highlighted the urgency of new nAMD treatments: “Up to 46% of AMD patients respond poorly or develop resistance to anti-VEGF therapies. AMD patients deserve safe, effective treatment options. The BRIGHT trial will evaluate HG202’s safety and efficacy to address this gap. We look forward to enrolling patients soon.” “Our AI/ML-driven HG-PRECISE® platform led us to discover the Cas13X/Y system ( Nature Methods 2021),” added Hui Yang, Ph.D., Co-Founder and Chief Scientific Advisor of HuidaGene. “Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects ( Nature Biotechnology 2022), laying out the technical foundation for future clinical applications.” BRIGHT Trial of HG202 (NCT06623279) The BRIGHT (an open-la B el, dose-escalation study for CRISPR/Cas13- R NA target I n G t H erapy for the T reatment of neovascular AMD) trial, a Phase 1, dose-finding study, will investigate the safety and efficacy of HG202 at different doses in nAMD patients. Primary endpoints include the safety and tolerability of HG202, with secondary endpoints assessing improvements in visual acuity, retinal thickness, and the need for anti-VEGF injections. About HuidaGene HuidaGene utilizes its proprietary CRISPR-based HG-PRECISE® platform to develop potentially curative genome medicines. The Company is advancing clinical programs, including trials of HG004 (granted ODD & RPDD by FDA) ‘ LIGHT ‘ trial (NCT06088992) and Phase 1/2 international, master-protocol ‘ STAR ‘ clinical trial (NCT05906953) for RPE65 -associated retinal disease, HG202 RNA-editing therapy ‘ SIGHT-I ‘ first-in-human trial (NCT06031727) and ‘ BRIGHT ‘ Phase 1 clinical trial (NCT06623279) for nAMD, HG204 RNA-editing therapy (granted ODD & RPDD by FDA and ODD by EMA) ‘ HERO ‘ trial (NCT06615206) for MECP2 duplication syndrome, and HG302 DNA-editing therapy (granted ODD & RPDD by FDA) first-in-human ‘ MUSCLE ‘ trial (NCT06594094) for DMD. The preclinical programs include HG303 DNA-editing for ALS and CRISPR RNA-editing therapy for Alzheimer’s. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn . SOURCE: HuidaGene Therapeutics

Global clinical-stage biotech company HuidaGene Therapeutics has received FDA clearance to move forward with its application for its investigational drug, a CRISPR/Cas13 RNA-editing therapy to treat neovascular age-related macular degeneration (nAMD), dubbed HG202, developed using machine learning and AI. The company used its HG-PRECISE platform (HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) to devise the therapy. HG-PRECISE allows for the quick discovery of Cas proteins using AI and machine learning in DNA sequencing. It also provides assembly predictions from a metagenomic database. The company will move forward with a phase one study to evaluate HG202's effectiveness at different doses in patients with nAMD. The company says the BRIGHT trial will begin enrolling patients soon. "Our AI/ML-driven HG-PRECISE platform led us to discover the Cas13X/Y system," Hui Yang, cofounder and chief scientific advisor of HuidaGene, said in a statement. "Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects, laying out the technical foundation for future clinical applications." THE LARGER TREND Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, developed more than a decade ago, allows scientists to modify DNA sequences of living organisms. Potential applications range from treatments for genetic disorders to researching disease mechanisms. Gene editing technologies, such as SHERLOCK and DETECTR, are transforming digital diagnostics, enabling rapid detection of infectious diseases like COVID-19. Still, ethical, legal and scientific factors warrant consideration, and instabilities can happen that can be fatal for someone, according to experts. Gene editing can cause irreversible changes, and changes to a DNA sequence can last for generations. Another company working with CRISPR technology is AI-enabled protein design company Profluent, which has leveraged AI to design an open-source gene editor called OpenCRISPR-1, demonstrating the technology can be used to create molecules with the power to edit human DNA.

HG202 is the first CRISPR/Cas13Y RNA-targeting therapy to enter clinical development and the only clinical-stage CRISPR RNA-editing therapy for age-related macular degeneration Preclinical studies in laser-induced CNV mice showed HG202 reduced CNV area by 87%, outperforming both anti-VEGF antibodies and AAV-anti-VEGF gene therapy SHANGHAI and CLINTON, N.J., Nov. 4, 2024 /PRNewswire/ -- HuidaGene Therapeutics ("HuidaGene"), a global clinical-stage biotechnology company, announced that the U.S. FDA has cleared its investigational new drug (IND) application for HG202, the world's first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD), which affects millions worldwide. "This open IND for HG202 by the U.S. FDA-the first regulator to clear CRISPR/Cas13 for clinical development–marks a significant milestone for HuidaGene and the CRISPR RNA-editing field," noted Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. "The strong preclinical data for HG202, combined with promising results from the first-in-human ' SIGHT-I' trial in China, underscored its potential to address AMD using a non-receptor binding pathway through the Cas13 RNA editor to knockdown VEGF-A mRNA." Xin Zhang, M.D., MSc., COO and CMO of HuidaGene, highlighted the urgency of new nAMD treatments: "Up to 46% of AMD patients respond poorly or develop resistance to anti-VEGF therapies. AMD patients deserve safe, effective treatment options. The BRIGHT trial will evaluate HG202's safety and efficacy to address this gap. We look forward to enrolling patients soon." "Our AI/ML-driven HG-PRECISE® platform led us to discover the Cas13X/Y system (Nature Methods 2021)," added Hui Yang, Ph.D., Co-Founder and Chief Scientific Advisor of HuidaGene. "Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects (Nature Biotechnology 2022), laying out the technical foundation for future clinical applications." BRIGHT Trial of HG202 (NCT06623279) The BRIGHT (an open-la Bel, dose-escalation study for CRISPR/Cas13- RNA target In G t Herapy for the Treatment of neovascular AMD) trial, a Phase 1, dose-finding study, will investigate the safety and efficacy of HG202 at different doses in nAMD patients. Primary endpoints include the safety and tolerability of HG202, with secondary endpoints assessing improvements in visual acuity, retinal thickness, and the need for anti-VEGF injections. About HuidaGene HuidaGene utilizes its proprietary CRISPR-based HG-PRECISE® platform to develop potentially curative genome medicines. The Company is advancing clinical programs, including trials of HG004 (granted ODD & RPDD by FDA) ' LIGHT' trial (NCT06088992) and Phase 1/2 international, master-protocol ' STAR' clinical trial (NCT05906953) for RPE65-associated retinal disease, HG202 RNA-editing therapy ' SIGHT-I' first-in-human trial (NCT06031727) and ' BRIGHT' Phase 1 clinical trial (NCT06623279) for nAMD, HG204 RNA-editing therapy (granted ODD & RPDD by FDA and ODD by EMA) ' HERO' trial (NCT06615206) for MECP2 duplication syndrome, and HG302 DNA-editing therapy (granted ODD & RPDD by FDA) first-in-human ' MUSCLE' trial (NCT06594094) for DMD. The preclinical programs include HG303 DNA-editing for ALS and CRISPR RNA-editing therapy for Alzheimer's. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn. SOURCE HuidaGene Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED