Gritgen Therapeutics Co., Ltd

SUZHOU, China I November 16, 2023 I Gritgen Therapeutics Co., Ltd. (Gritgen), a biotechnology company focused on identifying and developing innovative gene therapy products to achieve "Great Science for Human Health", announced that it has successfully completed enrollment of all 12 patients in the Investigator Initiated Trial (IIT) by May 17, 2023 for GS1191, an innovated gene therapy product for Hemophilia A (HA) treatment. GS1191, is now the first one completing patient enrollment for IIT study in China. Gritgen also announced that the first patient has been dosed in the China Phase I trial (Protocol No.: GS1191-0445-GTHA-CN01) evaluating GS1191 in patients with severe Hemophilia A. The IIT is a single-arm, open-label, single-dose administration clinical trial to evaluate the safety and tolerability of GS1191 in HA patients older than 18 years of age whose endogenous factor VIII (FVIII) activity was lower than 1 IU/dL (<1%). 12 patients with HA were enrolled in two dose groups (6 in the 2E12 vg/kg group and 6 in the 4E12 vg/kg group, respectively). The last patient was enrolled on May 17, 2023, and all the enrolled patients have been followed up for at least 12 weeks after dosing by Aug. 9, 2023. Preliminary results indicate that GS1191 can continuously and significantly improve the endogenous FVIII activity. All the enrolled patients have experienced significant reductions in Annualized Bleeding Rate (ABR) after receiving GS1191 infusions. Currently, all patients enrolled in 4E12 vg/kg dose group have normal FVIII activities with no episodes of bleeding events observed after dosing. Most of the adverse events (AEs) reported in this IIT study were grade 1 in severity. No AEs have been observed in any patients receiving either dose considered to be grade 3 or higher in severity, indicating a favorable safety profile. GS1191 received clinical trial approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) in January of this year. The IND approval was granted by NMPA in January 2023. This multicenter, single-arm, open-label, single-dose administration Phase 1 study was designed to evaluate the safety, preliminary efficacy, and vector kinetics profile of a single intravenous infusion of GS1191 at different dose levels in patients with severe HA and determine the dose for Phase 2/3 clinical studies based on the patients’ benefit/risk profile. The first patient has been dosed in the China Phase 1 trial on Aug. 2, 2023. Dr. Wu Fenglan, Co-founder and CEO of Gritgen, commented that, "as the first flagship gene therapy product developed independently by our company, Gritgen's GS1191 has successfully achieved First Patient In Phase I and all the enrollment in the IIT study. We appreciate the investigators, patients and their families as well as our own team, for bringing effective and valuable treatment to HA patients." GS1191 is the first gene therapy product for HA gaining IND approval in China. Dr. Wu Fenglan further remarked that, "the outstanding safety and efficacy data from IIT have instilled immense confidence in the entire team. Gritgen is committed to developing innovative gene therapy products, revolutionizing the quality of life of patients at the genetic level, and reshaping the clinical treatment pattern of hemophilia A." About Hemophilia Hemophilia is an X-linked recessive hereditary bleeding disorder. Epidemiology estimates approximately 120,000 patients with hemophilia in China. This disorder can be classified into Hemophilia A (HA) and Hemophilia B (HB). HA accounts for about 85% of all hemophilia patients, making it the most prevalent subtype. HA results from mutations in the gene encoding FVIII, causing a genetic deficiency of coagulation factor VIII. HB, constitutes about 15% of all hemophilia cases and is caused by mutations in the F9 gene, resulting in an genetic deficiency of coagulation Factor IX. Factor VIII is larger than factor IX, making it the more difficult to be delivered by gene therapy. The success of delivering Factor VIII means that technically, the same team should be able to deliver Factor IX. The primary manifestations of hemophilia are spontaneous bleeding or excessive bleeding following minor injuries. The standard clinical treatment for hemophilia involves replacement therapy with coagulation factors, requiring lifelong injection of proteins on a regular basis, which brings a significant economic burden to patients, their families, and societies. Gene therapy stands as a cutting-edge technology with the potential to offer a cure for hemophilia patients with a single injection. About GS1191-0445 Injection GS1191 is an investigational adeno-associated virus (AAV) gene therapy for the treatment of Hemophilia A. GS1191 can deliver human FVIII gene to patients with Hemophilia A in vivo and achieve long-term gene expression via a single dose of intravenous infusion, thereby raising and sustaining endogenous FVIII activity over the long term. As an innovative treatment, the goal is to achieve a therapeutic effect of "single-dose, long-term efficacy" by addressing the underlying cause and preventing bleeding events. GS1191 is the first gene therapy product receiving IND approval from NMPA in January 2023, and the first product completing patient enrollment for IIT in China. About Gritgen Founded in 2019 by Dr. Yi Rao, a leader of biomedical research in China, Gritgen is dedicated to apply “Great Science for Human Health”. The company has independently developed a globally leading highly efficient GritPAC and GritOcul platform. Combining fundamental understanding of genetics and biology of diseases with modern technologies in molecular biology, Gritgen has developed multiple product pipelines in rare and common diseases to fulfill the unmet medical needs of patients and improve the health of people in China and the world. SOURCE: Gritgen

SUZHOU, China--(BUSINESS WIRE)-- Gritgen Therapeutics Co., Ltd. (“Gritgen”), a biotechnology company focused on discovering and developing innovative gene therapy products, announces the successful validation and official launching of operations at its commercial GMP facility located in Suzhou Industrial Park. This achievement sets the stage for the production of Gritgen's phase III clinical and commercial product for hemophilia A (HA). The construction of Gritgen's GMP facility strictly adheres to the global cGMP standards, and complies with the regulatory guidelines of authoritative agencies including the NMPA, the FDA and the EMA. It is now a leading commercial facility for AAV-based products in China. This press release features multimedia. View the full release here: Gritgen Therapeutics Launches Commercial GMP Facility in Suzhou, China (Photo: Business Wire) The facility has 8,600 square meters, covering plasmid production and AAV production lines. The production lines are based on a mature and reliable upstream process with mammalian cell suspension, and downstream process with chromatography platform technology, equipped with disposable bioreactors of different scales, of which the largest scale reaches 1,000 liters, taking into account of the productivity and quality of manufacturing. The facility is equipped with pilot filling lines and commercial fill-finish lines to meet the need of drug product manufacture. The facility also has a quality control laboratory covering biochemical, physicochemical, molecular biological, and microbiological tests. Production lines in the facility are completely physically separated, which comprehensively controls the risk of cross-contamination, and lays a solid foundation for the global supply chain guarantee of innovative gene therapy products. Dr. Wu Fenglan, Co-founder and CEO of Gritgen, stated, “The validation and operational launch of our GMP facility represents a pivotal juncture for Gritgen, which provides an important vector foundation for the company to realize commercialization. At the present, a bottleneck of gene therapy is the manufacturing process, a challenge resonating globally. The production capacity of large-scale, high-quality, cost controllable vectors is an important driving force for the development of gene therapy industry." Embarking on the concept to establish a “China-based, world-leading” biotechnology company, Gritgen is actively venturing into the worldwide arena of clinical development and commercialization for a portfolio of flagship gene therapy products. Powered by industry-leading intense expertise, innovative technology platforms, and state-of-the-art facilities, the company is committed to introducing more first-in-class and best-in-class gene therapy drugs to global patients. [About Gritgen] Founded in 2019 by Dr. Yi Rao, a leader of biomedical research in China, Gritgen is dedicated to apply “Great Science for Human Health”. The company has independently developed globally leading highly efficient GritPAC and GritOcul platforms. Combining fundamental understanding of genetics and biology of diseases with modern technologies in molecular biology, Gritgen develops multiple product pipelines in rare and common diseases to fulfill the unmet medical needs of patients and improve the health of people in China and the world. View source version on businesswire.com: Contacts Gritgen Therapeutics Co., Ltd. PR Manager Jiaojiao Xu jiaojiao.xu@gritgen.com Source: Gritgen Therapeutics Co., Ltd. Smart Multimedia Gallery Photo Gritgen Therapeutics Launches Commercial GMP Facility in Suzhou, China (Photo: Business Wire) Logo View this news release and multimedia online at: