Recently,SimbayPark FTIH Park EnterpriseJuventasAnnounce,Juventas' Self-Developed Innovative Product HY001N Cell Injection Receives NMPA CDE's Implicit Approval for IND Application to Treat Autoimmune Hemolytic Anemia (AIHA) Patients Who Failed at Least Three Prior Lines of TherapyThe clinical trial is led by Professor Shi Jun from the Blood Disease Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences) as the principal investigator. This is the world's first registered clinical trial using CAR-T cell therapy products with rapid preparation technology to treat autoimmune hemolytic anemia, bringing new hope to patients with refractory/relapsed AIHA. It also highlights Juventas' core breakthroughs in cell therapy technology innovation and its strategic layout in autoimmune diseases.
Autoimmune Hemolytic Anemia (AIHA) is a group of hemolytic anemias characterized by abnormal immune function leading to hyperactive B lymphocytes producing antibodies against the body's own red blood cells. The red blood cells adsorb autoantibodies and/or complements, causing accelerated destruction of red blood cells and affecting the patient’s lifespan. If a patient concurrently has ≥2 types of immune-mediated cytopenias, it is referred to as Evans syndrome.Currently, there is no epidemiological data on AIHA in China. Foreign data shows that the annual incidence rate of AIHA is (0.8-3.0)/100,000. AIHA can occur at any age, but most patients are over 40 years old, with the peak age of onset being 70-80 years old. There are more female patients than male patients.HY001N Cell Injection is the first product developed on Juventas' innovative rapid preparation NexT technology platform. Compared with traditional autologous CAR-T technology platforms, the rapid preparation NexT process reduces the in vitro expansion and culture steps of CAR-T cells, shortening the production time in the workshop from 9-14 days to less than 2 days. Patient waiting time is reduced by 50%, or up to 10 days, while production costs are significantly decreased compared to existing preparation technologies. Meanwhile, the proportion of T naïve cells in the final rapid preparation CAR-T product is significantly increased, laying a foundation for its efficacy and safety.Chief Physician of the Hematology Hospital, Chinese Academy of Medical Sciences:
HY001N Cell Injection Receives Implied Approval, Marking a Significant Breakthrough in the Treatment of Refractory Autoimmune Hemolytic Anemia (AIHA). From a clinical perspective, about 50% of AIHA patients still experience relapse or long-term steroid dependence after multiple lines of treatment, with a disability and mortality risk as high as 20%. Current chemical drug treatments have shown limited efficacy for these patients, while the emergence of innovative CAR-T cell therapy products offers them a new treatment option.
Chief Clinical Expert of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences; Director of the National Clinical Research Center for Hematological Diseases:
HY001N Cell Injection Receives Implied Permission: This is a key expansion into the field of autoimmune hemolytic anemia for the CAR-T product based on the NexT rapid preparation technology, and also an important academic breakthrough in cell therapy for autoimmune diseases. The clinical need for refractory AIHA is urgent, with significant limitations in existing treatments. It is hoped that this product will validate the application value of cell therapy in autoimmune diseases through scientifically rigorous clinical research, providing new clinical options for the treatment of such conditions.
CEO of Juventas:
We are very pleased that the IND for HY001N cell injection has received tacit approval from the Center for Drug Evaluation of the National Medical Products Administration. This marks an important milestone in the company’s development of innovative technology platforms. HY001N is a key product on Juventas' rapid CAR-T manufacturing technology platform and will bring new breakthrough treatment options to more patients with autoimmune diseases, including AIHA. Building on the approval and market launch of its core product, Nacioran Injection, for diseases such as leukemia and lymphoma, Juventas has established an internationally leading innovative cell drug R&D system, quality control system, and production system, including autologous CAR-T cell therapy technology, rapid manufacturing technology, universal cell technology, and in vivo generation technology platforms. The company continuously reduces costs and improves drug accessibility through technological innovation, benefiting more patients.
Juventas, founded in June 2018, has grown into a leading innovator in China's cell therapy industry, committed to becoming a globally leading biopharmaceutical enterprise driven by cutting-edge cell and gene technologies.
The Company's First Core Product: Yuan Ruida®(Najiolun Injection, CNCT19) was officially approved for marketing by the National Medical Products Administration in November 2023. This product is the first CAR-T drug in China's leukemia treatment field and also the first fully self-innovated CD19 CAR-T drug in China. Juventas has deeply cooperated with top national institutions to build an international new drug research and development system centered on technology platforms such as the CAR technology platform and gene editing technology platform. It has more than 10 pipeline products under research covering diseases such as hematological tumors, solid tumors, and autoimmune diseases. Yuan Ruida.®(Inaticabtagene Autoleucel Injection, CNCT19) is a CAR-T cell therapy product with proprietary intellectual property that targets CD19. It features a globally unique CD19 scFv (HI19a) structure and internationally leading manufacturing processes. It has successively obtained three Investigational New Drug (IND) approvals from the National Medical Products Administration (NMPA) for the treatment of adult relapsed or refractory acute lymphoblastic leukemia (r/r B-ALL), relapsed or refractory aggressive B-cell non-Hodgkin lymphoma, and pediatric and adolescent relapsed or refractory B-cell acute lymphoblastic leukemia. The product has also been granted "Breakthrough Therapy Designation" by the Center for Drug Evaluation of the NMPA and Orphan Drug Designation (ODD) by the U.S. FDA. In December 2022, the NMPA officially accepted the New Drug Application (NDA) for Inaticabtagene Autoleucel Injection for the treatment of adult r/r B-ALL and included it in the priority review process. In March 2023, the IND application for Inaticabtagene Autoleucel Injection for the treatment of adult r/r B-ALL was approved by the U.S. FDA. In November 2023, Juventas...®(Juventas' Naciorl Injection) Officially Approved for Marketing by China's National Medical Products Administration (NMPA).
Juventas adheres to the direction of meeting clinical needs, and through a strict cell therapy product manufacturing and quality system, provides patients with safe, efficient, and accessible immune cell therapy products. The company possesses world-class R&D technology platforms, process development platforms, quality control systems, and commercial production bases, and obtained Tianjin's first-ever "Drug Production License" for cell-based drugs in June 2021. The company holds multiple invention patents and has been selected for the National Key Research and Development Program under the Ministry of Science and Technology, specifically the "Science and Technology Supporting the Economy 2020 Key Special Project." It has also received titles such as "National Intellectual Property Advantage Enterprise" and "Tianjin Specialized, Precise, Unique, and Innovative Small and Medium-sized Enterprise Recognition," among others.
Shanghai FT1 Life Science Park (Abbreviation"Simbay Park, Star North Free Trade Zone No.1 Project" is located in the Waigaoqiao Bonded Area, the core of Shanghai's Pudong New Area Free Trade Zone. It is an innovative service-oriented characteristic industrial park under the Star North Group, focusing on the incubation, cultivation, and transformation of medical enterprises.The park relies on the unique advantages of an outward-oriented economic zone with "Free Trade + Bonded" characteristics. By gathering the full range of service resources in the biomedical CRO+CDMO+CSO+EHS industry chain, government innovation and entrepreneurship support policies, and numerous well-known investment institutions, it provides enterprises with deep technical incubation in the medical industry, acting as a steward for the transformation of corporate technological achievements. It is committed to building an international biomedical industry innovation and entrepreneurship base.
