Lubs X-Linked Mental Retardation Syndrome

HG302 effectively generates stable genome editing with minimal off-target effects in-vitro and in-vivo A single intravenous injection of HG302 restores functional dystrophin protein expression HG302 demonstrates therapeutic effects by improving muscle functions in humanized DMD mice SHANGHAI and CLINTON, N.J., Dec. 19, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因"HuidaGene") today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. Continue Reading Unlike the in-vivo use of Cas9 system with limited genome-editing efficiency, high occurrence of off-targeting modifications, viral delivery packaging limitations, and complicated CRISPR-Cas9 patent dispute, HuidaGene identified novel Cas12i proteins using AI and deep machine-learning of DNA sequencing through the proprietary HG-PRECISE ® platform to develop high-fidelity Cas12i variant (hfCas12Max®). This hfCas12Max® is smaller in size than Cas9, thereby packaging it using one viral vector with the lowest off-targeting activity and highest editing efficiency in mammalian cells when compared to SpCas9 and LbCas12a. Furthermore, Cas12i was granted a patent by USPTO with a cleaner commercial landscape. HuidaGene Announces RPDD Granted to HG302, A Novel CRISPR DNA-editing Therapy, for the Treatment of DMD Post this "This is Company's third program receiving RPDD, suggesting the recognition of the importance of DMD and the preclinical safety and efficacy of hfCas12Max gene-editing therapy by FDA," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and CEO of HuidaGene. "DMD patients are often wheelchair-bound before the age of 12 facing a significant unmet need. HG302 is designed specifically to target the DMD gene and enable the production of functional dystrophin protein. Time is muscle; we look forward to advancing HG302 into the clinic as soon as possible." The RPDD is granted for rare medical diseases affecting fewer than 200,000 people in the US and primarily for individuals aged under 18. If the HG302 Biologic Licensing Application (BLA) for DMD is approved by the FDA, HuidaGene may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred this high-value PRV. PRVs have historically commanded prices over US$ 100 million. About HG302 HG302 is a novel CRISPR DNA-editing therapy with high-fidelity Cas12 (hfCas12Max) and CRISPR RNA targeting human DMD exon 51 splice-donor (SD) site delivered by a single viral vector into the muscles. The human DMD exon 51 SD site will be disrupted by HG302, thereby allowing exon 51 skipping to form the correct open reading frame from exons 50 to 53, restoring functional dystrophin protein expression and improving muscle functions. About HuidaGene - 辉大基 因 HuidaGene Therapeutics (辉大基因), a global clinical-stage biotechnology company, utilizes its CRISPR-based HG-PRECISE® platform to discover, engineer, and develop potentially curative genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted both ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including HG301 CRISPR DNA-editing in retinitis pigmentosa, HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted both ODD and RPDD), and HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted RPDD) etc. The Company's extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicine. Learn more at huidagene.com or on LinkedIn. SOURCE HuidaGene Therapeutics

[01-November-2023] HG004 is the first China-developed AAV gene therapy medicine to be evaluated in a multinational, master-protocol clinical trial for patients with inherited retinal dystrophies caused by mutations in RPE65 gene HG004 is also first China independently-developed gene therapy drug granted both ODD and RPDD by the U.S. FDA HG004 is Company's first product candidate from robust pipeline of ophthalmology and neurology advances into the clinic 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 ( ) and rare pediatric disease designation ( ) 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) 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 RPE65 gene (RPE65-IRDs), are considered to represent a phenotypic continuum of the same disease. The RPE65-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 RPE65-IRDs, other areas of development including speech, social skills, and behavior may also be delayed. About Multinational, Master-Protocol Clinical Trial of HG004 (NCT05906953; CTR20232920) 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 RPE65 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 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 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 RPE65 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 MECP2 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 or follow us on LinkedIn at

HG004 is the first China-developed AAV gene therapy medicine to be evaluated in a multinational, master-protocol clinical trial for patients with inherited retinal dystrophies caused by mutations in RPE65 gene HG004 is also first China independently-developed gene therapy drug granted both ODD and RPDD by the U.S. FDA HG004 is Company's first product candidate from robust pipeline of ophthalmology and neurology advances into the clinic SHANGHAI and CLINTON, N.J., Oct. 31, 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 ( ) and rare pediatric disease designation ( ) 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) 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 RPE65 gene (RPE65-IRDs), are considered to represent a phenotypic continuum of the same disease. The RPE65-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 RPE65-IRDs, other areas of development including speech, social skills, and behavior may also be delayed. About Multinational, Master-Protocol Clinical Trial of HG004 (NCT05906953; CTR20232920) 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 RPE65 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 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 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 RPE65 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 MECP2 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 or follow us on LinkedIn at View original content to download multimedia: SOURCE HuidaGene Therapeutics