CYP4V2

Results from dose-escalation study show safety and preliminary efficacy signal of NGGT001 for the treatment of BCD WALNUT CREEK, CA, USA I January 14, 2025 I NGGT Inc., (“Next Generation Gene Therapeutics” or “NGGT” or the “Company”), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced the publication in the journal JAMA Ophthalmology of the results from a dose-escalation study in patients with BCD, entitled “ Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy “.  The peer-reviewed article can be accessed here: Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy Bietti Crystalline Dystrophy (BCD) is a rare, inherited genetic eye disease that causes progressive vision loss, often leading to legal blindness. Caused by mutations in the CYP4V2 gene, BCD currently has no approved treatment, leaving patients with limited options and a bleak prognosis. This open-label, dose-escalation trial, conducted at two sites, involved 12 participants with genetically confirmed BCD. Participants received a single subretinal injection of either 1.5 e11 or 3.0e11 total vector genomes of a gene therapy called rAAV-hCYP4V2 (NGGT001), which delivers a functional copy of the CYP4V2 gene. Key findings of the trial: Safety: The trial found no substantial safety concerns associated with the gene therapy over 12 months of follow-up. Only one participant experienced mild intraocular inflammation, which resolved quickly. No severe adverse events related to the treatment or dose-limiting toxic effects were observed. Vision Improvement: While the trial was primarily designed to assess safety, encouraging improvements in vision were observed. At 12 months, the treated eye showed a mean improvement of 13.9 letters on the BCVA, compared with 6.3 letters in the untreated eye. Importantly, participants with residual autofluorescence (AF) in the fovea, a sign of remaining functional photoreceptors, showed sustained visual gains over the 12 months. These early findings suggest that gene therapy with rAAV-hCYP4V2 has the potential to be a safe and effective treatment for BCD. However, further research is needed to confirm these findings and to determine the long-term effects of the therapy. “We are thrilled to see the results from our dose-escalation trial, which indicate the promise of NGGT001 to serve as a safe treatment for BCD,” said Yiting Liu, Ph.D., VP of Translational Research at NGGT. “Across the 12-month span of patient follow-up, we observed promising indications of visual improvement following treatment with NGGT001 and no severe adverse events. We look forward to further clinical trials to validate NGGT001’s ability to provide a durable treatment for this debilitating inherited disease.” “Given the lack of effective treatments for BCD, we are extremely encouraged by these results,” said Lixin Jiang, M.D., Ph.D., CEO and Co-Founder of NGGT. “The impressive early safety profile of NGGT001 and sustained improvement in visual acuity in the study across patients strongly supports its potential for the treatment of BCD.” NGGT001 is an AAV-based gene therapy candidate designed to provide a functional copy of the CYP4V2 gene to restore enzymatic fatty acid metabolism. BCD is a severe autosomal recessive genetic disorder caused by a mutation in the CYP4V2 gene that causes metabolic enzyme dysfunction wherein yellow-white crystalline lipid deposits form in the retina and cornea, causing the retinal pigment epithelium to degenerate. Resultant symptoms include night blindness and decreased vision in the second to third decade of life, progressing to legal blindness. About NGGT Inc. NGGT is a clinical-stage biotechnology company developing novel gene therapies for the treatment of retinal, metabolic and neurodegenerative diseases. The Company is currently advancing an extensive therapeutic development pipeline with multiple clinical and pre-clinical programs built around several proprietary AAV (Adeno-Associated Virus) products: NGGT001, a Phase I/II-ready gene therapy for Bietti’s Crystalline Dystrophy; NGGT002, a Phase I/II-ready gene therapy for PKU; NGGT006, a gene therapy for familial hypercholesterolemia, currently undergoing Investigator-Initiated Trials; and NGGT007, a gene therapy for Wet-AMD. NGGT is headquartered in Walnut Creek, California. For more information, please visit www.nggtbio.com , contact info@nggtbio.com and follow us on X and LinkedIn . SOURCE: NGGT

Results from dose-escalation study show safety and preliminary efficacy signal of NGGT001 for the treatment of BCD WALNUT CREEK, Calif., Jan. 14, 2025 /PRNewswire/ -- NGGT Inc., ("Next Generation Gene Therapeutics" or "NGGT" or the "Company"), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced the publication in the journal JAMA Ophthalmology of the results from a dose-escalation study in patients with BCD, entitled " Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy". The peer-reviewed article can be accessed here: Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy Bietti Crystalline Dystrophy (BCD) is a rare, inherited genetic eye disease that causes progressive vision loss, often leading to legal blindness. Caused by mutations in the CYP4V2 gene, BCD currently has no approved treatment, leaving patients with limited options and a bleak prognosis. This open-label, dose-escalation trial, conducted at two sites, involved 12 participants with genetically confirmed BCD. Participants received a single subretinal injection of either 1.5 e11 or 3.0e11 total vector genomes of a gene therapy called rAAV-hCYP4V2 (NGGT001), which delivers a functional copy of the CYP4V2 gene. Key findings of the trial: Safety: The trial found no substantial safety concerns associated with the gene therapy over 12 months of follow-up. Only one participant experienced mild intraocular inflammation, which resolved quickly. No severe adverse events related to the treatment or dose-limiting toxic effects were observed. Vision Improvement: While the trial was primarily designed to assess safety, encouraging improvements in vision were observed. At 12 months, the treated eye showed a mean improvement of 13.9 letters on the BCVA, compared with 6.3 letters in the untreated eye. Importantly, participants with residual autofluorescence (AF) in the fovea, a sign of remaining functional photoreceptors, showed sustained visual gains over the 12 months. These early findings suggest that gene therapy with rAAV-hCYP4V2 has the potential to be a safe and effective treatment for BCD. However, further research is needed to confirm these findings and to determine the long-term effects of the therapy. "We are thrilled to see the results from our dose-escalation trial, which indicate the promise of NGGT001 to serve as a safe treatment for BCD," said Yiting Liu, Ph.D., VP of Translational Research at NGGT. "Across the 12-month span of patient follow-up, we observed promising indications of visual improvement following treatment with NGGT001 and no severe adverse events. We look forward to further clinical trials to validate NGGT001's ability to provide a durable treatment for this debilitating inherited disease." "Given the lack of effective treatments for BCD, we are extremely encouraged by these results," said Lixin Jiang, M.D., Ph.D., CEO and Co-Founder of NGGT. "The impressive early safety profile of NGGT001 and sustained improvement in visual acuity in the study across patients strongly supports its potential for the treatment of BCD." NGGT001 is an AAV-based gene therapy candidate designed to provide a functional copy of the CYP4V2 gene to restore enzymatic fatty acid metabolism. BCD is a severe autosomal recessive genetic disorder caused by a mutation in the CYP4V2 gene that causes metabolic enzyme dysfunction wherein yellow-white crystalline lipid deposits form in the retina and cornea, causing the retinal pigment epithelium to degenerate. Resultant symptoms include night blindness and decreased vision in the second to third decade of life, progressing to legal blindness. About NGGT Inc. NGGT is a clinical-stage biotechnology company developing novel gene therapies for the treatment of retinal, metabolic and neurodegenerative diseases. The Company is currently advancing an extensive therapeutic development pipeline with multiple clinical and pre-clinical programs built around several proprietary AAV (Adeno-Associated Virus) products: NGGT001, a Phase I/II-ready gene therapy for Bietti's Crystalline Dystrophy; NGGT002, a Phase I/II-ready gene therapy for PKU; NGGT006, a gene therapy for familial hypercholesterolemia, currently undergoing Investigator-Initiated Trials; and NGGT007, a gene therapy for Wet-AMD. NGGT is headquartered in Walnut Creek, California. For more information, please visit , contact [email protected] and follow us on X and LinkedIn. For more information: NGGT, Inc. 2910 Camino Diablo, Suite 120 Walnut Creek, CA 94597 [email protected] SOURCE NGGT Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SHANGHAI, April 29, 2023 /PRNewswire/ -- On April 21, 2023, OBiO Technology (Shanghai) Corp., Ltd. (OBiO) announced a strategic agreement of commercial manufacturing collaboration with CHINAGENE. Under the agreement, OBiO will provide holistic contract development and manufacturing services to CHINAGENE for their gene therapy products, including large-scale cGMP manufacturing service for clinical trials, process characterization and validation services, product formulation and filling services, and commercial manufacturing. In addition, OBiO and CHINAGENE will continue to expand the cooperation in academic communication globally, to promote the innovation of gene therapy. "This agreement signed by OBiO and CHINAGENE is a continuation and sublimation from previous cooperation," Guodong Jia, CEO of OBiO said, "CHINAGENE is a leading biotech among the ophthalmic gene therapy developer. OBiO is a leading gene and cell therapy CDMO in China. OBiO will continue to improve the capability of process development and cGMP management system, to promote the gene therapy product of CHINAGENE." "OBiO has abundant experience in gene therapy manufacturing and cGMP quality, management. We are happy to continue our collaboration to accelerate the commercialization of our ophthalmic gene therapy product ZVS101e." Xi Chen, the Vice President of CHINAGENE said, "In past, OBiO had helped us to get the investigational new drug (IND) approvals from National Medical Products Administration of China (NMPA) and the U.S. Food and Drug Administration (FDA). In future, we will continue to work closely with our own abilities, to help more patients. " On August 2021, ZVS101e, a gene therapy production from CHINAGENE, was authorized by the U.S. Food and Drug Administration (FDA) as an orphan drug. Data from an exploratory clinical trial (IIT) of ZVS101e for bietti crystalline dystrophy (BCD disease) showed good safety and efficacy. In December 2022, ZVS101e was approved to conduct the Investigational New Drug (IND approval) clinical trials for the treatment of crystallization-like retinitis pigmentosa caused by CYP4V2 biallelic mutations, by NMPA and FDA. About CHINAGENE TECH CHINAGENE TECH is a developer of clinical genetic diagnosis, fertility prevention and gene therapy drug for hereditary eye diseases. The company specializes in developing ophthalmic gene therapy drugs backed by accurate gene diagnosis, providing reliable ophthalmic gene pharmaceuticals for the public. About OBiO Technology OBiO Technology (Shanghai) Corp., Ltd. (OBiO, SSE:688238.SH) is a gene and cell therapy-focused biotechnology company founded in 2013. In China for global customers, we offer holistic research, development, and manufacturing solutions for vectorology studies, functional genomics, process and analytical development, IND-enabling CMC, clinical and commercial manufacturing. With "enable gene therapy for better lives" as our mission, we are committed to providing high-quality service to global customers, advancing your product from bench to clinic, and bringing benefit to populations around the world. For more information about OBiO, visit . In the 26th annual meeting of American Society for Gene and Cell Therapy (ASCGT), OBiO will present the progress of in-house discovery regarding noval gene vectors, and advanced process development. OBiO will hold 2 oral presentations and 5 posters in the meeting. Welcome to OBiO's booth (#747) to get more information or attend our oral presentation in room 515AB on May 16 th 1:45pm to 2:00pm，other one is room 511 on May 17th. We also have 5 posters (#1644, #1445, #940, #810) to show our innovation. SOURCE OBiO Technology (Shanghai) Corp., Ltd.