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According to incomplete statistics, in January 2024, there were 9 CGT therapiesINDObtainThe batch involves 9 companies including Gracell, Oricell Therapeutics, B&G Bio, and IASO Bio. The editor has compiled the list as follows:
On January 3, 2024, KL003 Cell Injection, independently developed by Kanglin Biotechnology (Hangzhou) Co., Ltd., successfully obtained the clinical trial implied permission from the National Medical Products Administration (NMPA) of China.
KL003 Cell Injection is a gene-modified autologous hematopoietic stem cell product clinically used to treat transfusion-dependent β-thalassemia in adults or children. The product involves the transduction of autologous hematopoietic stem cells with a lentiviral vector carrying the β-globin gene, followed by reinfusion into the patient. This leads to the differentiation of red blood cells expressing functional β-globin, restoring the patient’s hemoglobin levels, eliminating the need for transfusions, and achieving a one-time functional cure.January 15, 2024According to the announcement on the CDE official website, the third globally pioneering product pipeline of Gracell, BRL03 Injection, has been approved for clinical trials to treat various advanced solid tumors, including lung cancer and gastric cancer.
On September 9 last year, the U.S. FDA approved the IND application for BRL03's Phase I/II clinical trial, and the NMPA IND application for BRL03 injection was also approved, marking that Gracell has officially obtained the IND approval for its 7th globally first-in-class product. As the first TCR-T product independently developed by Gracell to enter clinical trials, IIT studies have shown that BRL03 injection demonstrates good safety and preliminary efficacy in the treatment of solid tumors.
January 16, 2024Lingyi Biotech Co., Ltd.Lingyi Biotech's self-developed Class I therapeutic biological product, LY-M001 Injection, has successfully obtained the tacit approval from the National Medical Products Administration (NMPA) for its Investigational New Drug (IND) application (Acceptance No.: CXSL2300730).
LY-M001 Injection is the first domestically developed AAV gene therapy drug in China targeting Type I or Type III Gaucher disease.This product uses recombinant adeno-associated virus (rAAV) as a vector, and after a single intravenous infusion, it can express the glucocerebrosidase required by patients. Lingyi Biotech has developed an improved gene therapy vector for glucocerebrosidase with fully independent intellectual property rights, which can achieve long-term stable expression in the body and degrade harmful glycolipid metabolites, thereby achieving the goal of long-term treatment of Gaucher's disease.
R&D Pipeline
January 17, 2024Non-genetically Modified Natural Killer Cell Injection Independently Developed by Shanghai Enke Cell Technology Co., Ltd. (Enke Pharmaceuticals)Project CodeNK010) Obtain the U.S. Food and Drug Administration (FDA）IPhase Clinical Trial (IND) license. Currently, this product is the first in China to receiveFDAApproved Non-Gene-Modified Allogeneic Peripheral BloodNKCellular Medicine.
NK010With the advantages of optimized receptor spectrum, diversified targets, high purity, and broad applicability, it has the potential to treat various types of tumors.NK010There is also potential space for expansion to the treatment of non-tumor diseases, as well as a series of subsequent syntheses.NKCellular Medicine (SynNK) The best chassis cell. This timeIThe first choice for Phase clinical trials is ovarian cancer indications, and preclinical studies have shown,NK010Cell injection has demonstrated potent antitumor activity in animal models of solid tumors such as ovarian cancer and liver cancer, as well as in acute myeloid leukemia.
January 23, 2024The CDE official website shows that the Investigational New Drug (IND) application for the PD-1 gene-edited T-cell injection developed by Mercer (Beijing) Biotechnology Co., Ltd. ("Mercer") has been approved. This product is an autologous T-cell preparation with PD-1 gene knockout, intended for late-stage non-small cell lung cancer patients aged 18-75.
The "PD-1 Gene-Edited T Cell Injection" that has been granted clinical tacit approval this time was developed and produced by Chengdu Mercer Biotechnology Co., Ltd. and submitted to the National Medical Products Administration (NMPA) through its wholly-owned subsidiary, Mercer (Beijing) Biotechnology Co., Ltd., as a novel cell-based drug.The First Cell Type I New Drug in Sichuan Province to Obtain IND Approval。
On January 25, 2024, IASO Bio announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) had officially approved the Investigational New Drug (IND) application (Acceptance No.: CXSL2300759) for its fully human BCMA-targeted chimeric antigen receptor autologous T-cell injection (Equecabtagene Autoleucel Injection, R&D code CT103A) to expand its indications to include refractory generalized myasthenia gravis (Myasthenia Gravis, MG).IkyOrelse Injection(Product name: Focux®) was approved for marketing by the National Medical Products Administration on June 30, 2023, for the treatment of relapsed and refractory multiple myeloma.The approval of this IND for myasthenia gravis further expands the indications range for IASO Bio's Equecabtagene Autoleucel Injection. This is the second autoimmune indication for which it has received IND approval, following Neuromyelitis Optica Spectrum Disorder (NMOSD). IASO Bio is the first company in China to apply CAR-T products for autoimmune indications, and it is expected to change the treatment landscape for autoimmune diseases.On January 26, 2024, the Investigational New Drug (IND) application for GMCN-508B, a gene therapy drug for β-thalassemia submitted by CNMDICR BIOPHARMA, received tacit approval from the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA), marking the company's official entry into the registered clinical trial phase.
The approval of this IND marks an important milestone for CNMDICR BIOPHARMA, an emerging company specializing in lentiviral vector and hematopoietic stem cell therapy, signifying its successful transformation from "scientists" to "pharmaceutical professionals."
On January 29, 2024, Oricell Therapeutics announced that the U.S. Food and Drug Administration (FDA) had officially approved OriCAR-017 for the treatment of patients with relapsed/refractory multiple myeloma (R/R MM).New Drug Clinical Application (IND)。
OriCAR-017 is a chimeric antigen receptor T-cell therapy (CAR-T) targeting GPRC5D developed by Oricell Therapeutics using its proprietary technology platforms. The design and development of this product integrate Oricell's self-innovated Ori®Ab antibody platform, Ori®CAR structure platform, and the company’s expertise in CMC. It has demonstrated robust and durable anti-tumor efficacy as well as excellent safety in early exploratory clinical studies. With this IND approval, Oricell Therapeutics is about to initiate the clinical development of OriCAR-017 in the United States.On January 30, the company announced that the U.S. Food and Drug Administration (FDA) had officially approved a new Investigational New Drug (IND) application for the FasTCAR-T GC012F therapy, allowing the company to initiate early-line treatment with GC012F in the United States.Multiple Myeloma(ELMM) Phase 1 Clinical Trial.GC012F is a BCMA/CD19 dual-targeting autologous CAR-T cell therapy developed based on Gracell's proprietary FasTCAR technology platform. It has the potential to bring about transformative, rapid, deep, and durable effects in cancer and autoimmune disease treatment, with differentiated safety advantages. Currently, the company is conducting multiple clinical studies on FasTCAR-T GC012F, covering indications for various hematologic malignancies and autoimmune diseases. Clinical data consistently demonstrate the drug’s excellent efficacy and safety across clinical trials. The company has initiated a Phase 1b/2 IND clinical trial in the United States to evaluate GC012F for the treatment of relapsed/refractory multiple myeloma (RRMM), and a Phase 1/2 IND clinical trial for the same indication is also set to begin in China. Additionally, both the U.S. FDA and China's NMPA have approved the IND application for GC012F targeting refractory systemic lupus erythematosus (rSLE); an investigator-initiated clinical trial for the same indication has also been launched. Recently, the U.S. FDA once again approved the IND application for GC012F as an earlier-line treatment for multiple myeloma.Official Websites of Each Company
