Heyuan Bio's CAR-T Therapy Inaticabtagene Autoleucel Receives World’s First IND Approval for Autoimmune Hemolytic Anemia

Apr 11, 2025 08:21 CST Updated 08:21

Juventas

Innovative Cell Therapy Drug Developer

2025Year4Month10Day, National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) Website public information shows: Juventas' Nalkylcyte Injection (trade name: Yuan Ruida^®) New Drug Clinical Trial Application for New Indications in the Field of Autoimmune Diseases (IND) Obtain tacit consent (Acceptance No.:CXSL2500030），Used to treat at least3Line treatment failure in autoimmune hemolytic anemia (AIHA）。This is the world's firstCAR-TThe product has received clinical implied permission for treatment.AIHA。It is also the successor to its2024Year10Monthly HarvestBatch Treatment of Refractory Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia (SLE-ITP) After,Naciorl Injection inField of Autoimmune Disease TreatmentAcquiredThe2New Drug Clinical Trial Approval, is another milestone breakthrough in the field of autoimmune diseases.

Autoimmune Hemolytic Anemia(Autoimmune hemolytic anemia, AIHA)It is due to abnormal immune function in the body.,Lead toBHyperactive cell function produces antibodies against their own red blood cells.,Red Blood Cell Adsorption Autoantibodies and(Or)Complement,A group of hemolytic anemias that accelerate the destruction of red blood cells and shorten their lifespan. This disease not only affects the patient's physical health but may also trigger a series of complications, which can be life-threatening in severe cases.

In2024Year #66American Society of Hematology (ASH) Annual Meeting, a research team led by Professor Shi Jun from the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences (CAMS), presented an oral report on the ineffectiveness of various drug treatments.11Refractory CaseAIHAPatient AcceptanceCAR-TComplete clinical remission was achieved after cell therapy, andCAR-TCell-related adverse reactions were mild, and patients showed good tolerance. Currently, all patients are in treatment-free remission.

Juventas Nadofarcept Injection (Trade Name: Yuan Ruida)^®) is the first domestically produced product with full independent intellectual property rights in China.CD19 CAR-TCell therapy products. Already deployed in adults with relapsed or refractoryBAcute Lymphoblastic Leukemia, Relapsed or Refractory LargeBCellular Lymphoma, Pediatric Relapsed or RefractoryBAcute Lymphoblastic Leukemia and Multiple Types of Autoimmune Diseases. Its treatment for adult relapsed or refractoryBNew Drug Application for Acute Lymphoblastic Leukemia Cells2023Year11Received official approval for marketing from the National Medical Products Administration (NMPA) in China.

”

Professor Shi Jun

Professor Shi Jun, Deputy Director of the Institute and Vice President of the Blood Disease Hospital of the Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences), and Director of the Red Blood Cell Disease Diagnosis and Treatment Center:

Autoimmune Hemolytic Anemia (AIHA) So far, it is still a relatively difficult disease to cure, with50%The patients belong to refractory cases./Relapse, and currently there are no clear therapeutic drugs or effective salvage treatment options available, leading to a treatment dilemma. We are very pleased to see the approval of the clinical trial for the new drug Naciorlenzumab for the treatment of autoimmune hemolytic anemia, which means that this product is expected to bring new treatment options to a wide range of patients.

”

Professor Wang Jianxiang

Blood Disease Hospital, Chinese Academy of Medical Sciences(Institute of Hematology, Chinese Academy of Medical Sciences)Chief Clinical Expert and Director of the National Clinical Research Center for Hematological Diseases, Professor Wang Jianxiang：

Sincerely congratulate China's self-developedCD19 CAR-TDrug Name: Naciorlen Injection (Yuan Ruida)^®) Treatment of Autoimmune Hemolytic Anemia (AIHA) New Drug Clinical Trial Application Approved! The injectable drug, Naciorlen, has already demonstrated breakthrough efficacy in the treatment of blood diseases such as leukemia and lymphoma. The approval of this new drug clinical trial for autoimmune hemolytic anemia marks another significant step forward in its application for treating autoimmune diseases. We look forward to Naciorlen Injection making positive progress in exploring more indications, benefiting more patients and advancing the field.CAR-TTherapies play a role in a broader range of disease areas.

”

Dr. Lulu Lyu

CEO of Juventas, Dr. Lulu Lv:

Juventas Naoki Oronase Injection (Trade Name: Yuan Ruida)^®) The expansion into new indications in the field of autoimmune diseases once again demonstrates the representation of Najiolumab Injection.CAR-TThe wide applicability of cell therapy across multiple disease areas. This product has demonstrated significant efficacy and good safety in clinical practices for indications such as leukemia, lymphoma, and autoimmune diseases, showing excellent therapeutic potential and clinical value. The team will continue to collaborate with all parties to ensure the smooth progress of the registration clinical trials. We will also steadily advance the research and development and clinical trials of multiple indications and subsequent products, bringing more Chinese innovative drugs with higher safety, better efficacy, and greater accessibility to patients.

”

About Juventas

Juventas, founded in June 2018, has grown into a leading innovator in China's cell drug industry, striving to become a globally leading biopharmaceutical company driven by cutting-edge cell and gene technologies. The company’s first core product, Yuanruida® (Nabasagene Celoleucel Injection, CNCT19), was officially approved for marketing by the National Medical Products Administration in November 2023. This product is China's first CAR-T drug in the field of leukemia treatment and also the first fully self-innovated CD19 CAR-T drug developed in China. Juventas collaborates closely with top-tier national research institutions and has established an international new drug R&D system centered on core platforms such as the CAR technology platform and gene-editing technology platform. The company has over 10 pipeline products under development, covering diseases such as hematological malignancies, solid tumors, and autoimmune disorders.

Yuan Ruida® (Nadofaragene Firadenovec Injection, CNCT19, Inaticabtagene Autoleucel Injection) is a CAR-T cell therapy product with proprietary intellectual property rights targeting CD19. It features a globally unique CD19 scFv (HI19a) structure and internationally leading manufacturing processes. It has successively obtained three New Drug Clinical Trial Approvals (IND) from the National Medical Products Administration (NMPA) for the treatment of adult relapsed or refractory acute lymphoblastic leukemia, relapsed or refractory aggressive B-cell non-Hodgkin lymphoma, and pediatric and adolescent relapsed or refractory B-cell acute lymphoblastic leukemia. It has also received the "Breakthrough Therapy Designation" from the Center for Drug Evaluation of the NMPA and Orphan Drug Designation (ODD) from the U.S. FDA. In December 2022, the NMPA officially accepted the New Drug Application (NDA) for Nadofaragene Firadenovec Injection for the treatment of adult r/r B-ALL and included it in the priority review. In March 2023, the IND application for Nadofaragene Firadenovec Injection for the treatment of adult r/r B-ALL was approved by the U.S. Food and Drug Administration (FDA). In November 2023, Yuan Ruida® (Nadofaragene Firadenovec Injection) was officially approved for marketing by the China National Medical Products Administration (NMPA).

Guided by clinical needs, the company is committed to creating safe, effective, and accessible immune cell therapy products for patients through a strict production and quality system for cell therapy products. Juventas possesses world-class R&D technology platforms, process development platforms, quality control systems, and commercial-scale production bases. In June 2021, it became the first company in Tianjin, China to obtain a "Pharmaceutical Production License" for cellular drugs. The company owns multiple invention patents and has been selected for the National Key Research and Development Program of the Ministry of Science and Technology of China under the "Science and Technology Supporting the Economy 2020 Key Special Projects." It has also received titles such as "National Intellectual Property Advantage Enterprise" and "Tianjin Specialized, Refined, Unique, and Innovative SME Certification."

E.N.D

Previous article recommendations:(Click the article title below to open the original text)

China's First AAV Gene Drug Approved for Marketing!

CDE Releases "Common Issues and Responses on the Chemistry of Recombinant Adeno-Associated Virus Vector In Vivo Gene Therapy Products for Marketing Application (Draft for Comments)"

Construction of a GMP Facility for Cell Drug Production in China Officially Begins

Maximum Single Award of 30 Million Yuan, Focusing on Cell Drugs, Nucleic Acid Drugs, Protein Drugs and Other Directions, Shenzhen Fully Supports Pharmaceutical R&D and Introduction

Promoting High-Quality Development of Innovative Pharmaceuticals like Cell and Gene Therapy, Beijing Municipal Medical Security Bureau and Other Nine Departments Issue New Policies

A Cell Therapy Company in China Completes Pre-A Round Financing

A Brief Analysis of Cell Culture Process Elements: The "Quality" and "Permeability" of Cell Culture Bags

Chinese Team Publishes in Cell, Reveals Optimal Timing for Stem Cell Infusion and New Strategy to Prevent aGVHD

Children's Hospital of Zhejiang University Officially Launches CAR-NK Therapy Project for Lupus Erythematosus! Two Pediatric Patients Have Safely Discontinued Immunosuppressants

Total investment of 2.5 billion yuan, construction area of 220,000 square meters, focusing on cell and gene therapy, a leading cell and gene industry transformation base in China officially inaugurated.

Total Investment of 120 Million Yuan! Juventas, a Biotech Company Focused on Cell Therapy and Gene Technology Translation, Opens in China

China's First Solid Tumor Cell Drug Marketing Application Accepted

China's National Health Commission Releases New Regulations on Human Genetic Resource Management: Biological Samples Such as Serum and Plasma Containing Minimal Residual or Free Cells or Genes Are No Longer Included in the Scope of Human Genetic Resource Materials Management

Investment of no less than 200 million yuan, the first industrialized cell production base in Hainan lands

A Ready-to-Use NK Cell Therapy Developed in China Approved for Clinical Trials

An RNA Drug Approved for Marketing

A Cell Gene Therapy Drug Produced in China Approved for Marketing in Macao

Chengnuo Medical's Circular RNA Therapy for Liver Cancer Published in "Molecular Cancer," Bringing New Hope for Liver Cancer Treatment!

World's First! iPS Cell Therapy Enables Paralyzed Patient to Stand Again

Publication of the Charge Standards for the First Batch of Cell Therapy Projects in Boao Lecheng, Hainan

Statement: InternalVolumeComeSourceFrom Juventas Official, ThisWenPurposeInKnowDisclaimer: All content is for reference only and does not constitute any advice.