Bietti Crystalline Dystrophy

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

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria WALNUT CREEK, CA, USA I November 11, 2024 I NGGT Inc., (“Next Generation Gene Therapeutics” or “NGGT”), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced positive new data on NGGT002, a clinical-stage gene therapy candidate for the treatment of Phenylketonuria (PKU). “We are pleased with the results from our investigator-initiated trial, as they show great promise for NGGT002 to serve as a safe and effective treatment for PKU,” said Yiting Liu, Ph.D., VP of Translational Research at NGGT. “With our initial 40-week span of positive results, we are optimistic that our Phase I/II clinical trials will further elucidate NGGT002’s potential to treat this rare and debilitating disease in a lasting manner.” “Given the difficulties associated with dietary restrictions for PKU and the potential benefits to patients from effective and durable reductions of phenylalanine levels, we view these results with great excitement,” said Lixin Jiang, M.D., Ph.D., CEO and Co-Founder of NGGT. “With the impressive safety and efficacy shown by NGGT002 in the investigator-initiated trial, we are enthusiastic about the upcoming results from our ongoing clinical trials in the United States and China.” The data, presented at the National PKU Alliance biennial conference in July, highlight the results from an investigator-initiated trial (IIT) conducted in China and demonstrate the safety and efficacy of NGGT002 for the treatment of PKU. PKU is a rare genetic disorder caused by a mutation in the phenylalanine hydroxylase (PAH) gene that causes an elevated blood level of phenylalanine and results in progressive intellectual and behavioral impairments, skin rashes, movement disorders and seizures. Five out of six patients receiving a high dose of NGGT002 in the study demonstrated a level of plasma phenylalanine within the normal range after three weeks of drug administration. The first patient dosed with NGGT002 in the IIT trial showed a persistent plasma phenylalanine level within the normal range 40 weeks after the drug was dosed. Phase I/II clinical trials in the U.S. ( NCT06332807 ) and China ( NCT06061614 ) have been initiated to evaluate the safety and efficacy of NGGT002 in adult patients with PKU. First patients in both trials will be dosed based on the effective dose which has been proved in the IIT study. About NGGT002 Gene Therapy Clinical Study in Adult Classic PKU The U.S.-based clinical trial ( NCT06332807 ) is a Phase I/II, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002, an rAAV-based vector carrying a functional copy of the human PAH gene, in adult subjects with classic PKU. Participants in two dose cohorts will receive a single administration of NGGT002 and will be followed for efficacy for 1 year and safety and 5 years. Further information regarding this Phase I/II study, including eligibility and exclusion criteria, can be found on clinicaltrials.gov or by calling 801-261-2000. About NGGT Inc. NGGT is a clinical-stage biotechnology company developing novel gene therapies for the treatment of retinal, metabolic and neurodegenerative diseases. NGGT 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