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Disclaimer: Due to limited expertise, errors are inevitable, and some information may not be the most up-to-date. Comments pointing out any issues are welcome. This article is only an introduction to drugs related to healthcare and is not a recommendation of treatment plans (if involved); it does not constitute any investment advice.
According to incomplete statistics, 18 CGT therapies have been approved for IND in January-February 2024, with CAR-T therapy drugs becoming the mainstream among the CGT drugs approved for marketing. The approved drugs include 11 cell products, 5 gene therapy products, and 2 siRNA drugs. The details are compiled 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. This product involves the transduction of autologous hematopoietic stem cells with a lentiviral vector-mediated β-globin gene, which are then reinfused. In the patient's body, these cells differentiate into red blood cells expressing functional β-globin, restoring the patient’s hemoglobin levels, thereby eliminating the need for transfusions and achieving a one-time functional cure.January 15, 2024According to the announcement on the CDE official website, BioSyngen's third globally pioneering product pipeline, 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.NMPA IND Application for BRL03 Injection Approved, marking that BioSyngen has officially obtained its 7th global first.IND Clinical Approval for Innovative Products.As the first TC developed by BioSyngen to enter clinical trialsR-T Product, IIT Study Shows BRL03 Injection Has Good Safety and Preliminary Efficacy in Solid Tumor Treatment.
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 (acceptance number: CXSL2300730) from the National Medical Products Administration (NMPA) for its Investigational New Drug (IND) application.
LY-M001 Injection is China's first self-developed AAV gene therapy drug 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 a modified glucocerebrosidase gene therapy vector 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 for Gaucher's disease.
R&D Pipeline
January 17, 2024Non-gene-modified Natural Killer Cell Injection independently developed by Shanghai Enke Cell Technology Co., Ltd. (Enke Pharmaceuticals)Project CodeNK010`) obtained the approval from the U.S. Food and Drug Administration (`FDA）IPhase Clinical Trial (IND) license. Currently, this product is the first in China to obtainFDAApproved Non-Gene-Modified Allogeneic Peripheral BloodNKCellular drugs.
NK010With the advantages of optimized receptor spectrum, diversified targets, high purity, and broad applicability, it has the potential to treat multiple types of tumors.NK010There is also potential space for expansion to the treatment of non-tumor diseases, followed by a series of syntheses.NKCellular Drug (SynNK) The best chassis cell. This timeIThe first choice for Phase clinical trials is ovarian cancer indications. Preclinical studies have shown,NK010Cell injection has shown strong anti-tumor activity in animal models of solid tumors such as ovarian cancer and liver cancer, as well as acute myeloid leukemia.
Mercer (Beijing) Biotechnology Co., Ltd.
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 the PD-1 gene knocked out, 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 through its wholly-owned subsidiary, Mercer (Beijing) Biotechnology Co., Ltd. It is also a new Category I cell drug.The First Cell Type I New Drug Approved for IND in Sichuan Province。
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 the clinical trial of its fully human BCMA-targeted chimeric antigen receptor autologous T-cell injection (Equecabtagene Autoleucel Injection, R&D code CT103A) for the expanded indication of refractory generalized myasthenia gravis.IasoOrelse Injection(Product name: FocuCell®) was approved for marketing by the National Medical Products Administration on June 30, 2023, for the treatment of relapsed or refractory multiple myeloma.The approval of this IND for myasthenia gravis further expands the indications range for IasoBio's Idecabtagene Vicleucel Injection, marking its second autoimmune indication following the approval of Neuromyelitis Optica Spectrum Disorder (NMOSD). IASO Bio is the first company in China to apply CAR-T products for autoimmune indications, with the potential to transform 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) of 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 focused on 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 the recent 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 another 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 efficacy for 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 outstanding 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); meanwhile, 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.On February 2, 2024, Suzhou Hepa Thera Biotech Co., Ltd. (hereinafter referred to as "Hepa Thera") announcedThe clinical application of its newly developed hepatitis B drug HT-101 (siRNA) has been approved by the U.S. FDA to directly target chronic hepatitis B virus infection.IbPeriodClinical Trial。HT-101 Injection is the first domestically produced anti-hepatitis B siRNA product to enter the clinical stage in China., all studies on healthy volunteers in China have been completed, as well as all dosing for the Phase Ib clinical trial on patients with chronic hepatitis B. Preliminary research data indicate that HT-101 demonstrates good safety and tolerability in both healthy populations and patients with chronic hepatitis B, and can significantly reduce hepatitis B surface antigen (HBsAg) in patients with chronic hepatitis B.HBsAg), The HBsAg reduction in the medium-high dose group reached more than 2lg without significant rebound.On February 7, according to the CDE official website,CHIGENOVO Co., Ltd.The "ZVS203e Injection" developed by CHIGENOVO Co., Ltd. has received clinical trial implied permission for the treatment of Retinitis Pigmentosa (RP) patients carrying the RHO-R135W (RHO c.403C>T) mutation.
It is reported that ZVS203e Injection is a gene-editing therapeutic drug that utilizes the third-generation artificial endonuclease CRISPR/Cas9 to perform targeted editing on the mutation hotspot of the RHO gene, achieving a one-time administration for lifelong cure. The drug received FDA Orphan Drug Designation in 2022, and related research findings were published in the internationally renowned comprehensive biology journal eLife in June 2023. On December 20, 2023, ZVS203e Injection, developed by CHIGENOVO, obtained the U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) approval by default.
February 7,SimnovaNK Cell Injection, Genetically Modified with Chimeric Antigen Receptor Targeting CD19 and Independently Developed, Officially Receives Clinical Tacit Approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for Moderate to Severe Refractory Systemic Lupus Erythematosus (SLE). This is an SLE indication.The first generic NK product in China has been approved for IND, and its clinical progress is even expected to become a global leader.
On February 7, Immunofoco announced its self-developed targetingThe clinical trial application for the autologous CAR-T cell injection product targeting EpCAM (IMC001) has received tacit approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration.(Application No.: CXSL2300792), for the treatment of EpCAM-positive advanced digestive system tumors, including but not limited to advanced gastric cancer (GC)/gastroesophageal junction adenocarcinoma (GEJ).
IMC001 BecomesThe world's first CAR-T product targeting EpCAM to receive IND approval; IMC001 has demonstrated good safety and efficacy in IIT studies.
2On the 23rd, Immunofoco announced that its self-developed autologous CAR-T cell injection product targeting EpCAM (IMC001) has recently been granted clinical research approval by the U.S. FDA after its clinical trial application was approved by China's CDE, for the treatment of EpCAM-positive advanced digestive system tumors, including but not limited to advanced gastric cancer (GC)/gastroesophageal junction adenocarcinoma (GEJ).
Immunotech Applied Science Limited
On February 18, Immunotech Applied Science Limited-B (06978.HK) successfully obtained clinical trial approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for its aT19 injection.aT19 Injection is a therapeutic biological product, classified as Class I biological product, with its main functional component being CD19 antigen-presenting T cells. The clinical trial protocol involves a Phase I study of sequential treatment using aT19 followed by CAR-T-19 injection for patients aged 25 years or younger with CD19-positive relapsed/refractory B-cell acute lymphoblastic leukemia. The proposed indication is for patients aged 25 years or younger with relapsed/refractory B-cell acute lymphoblastic leukemia who have achieved clinical benefit after CD19-targeted CAR-T cell therapy, aiming to enhance treatment efficacy and reduce recurrence. This trial is scheduled to commence within 2024.February 22,Jinweike BiotechnologyIndependently developed for neovascularizationAge-related Macular Degeneration(nAMD) Gene Therapy Product"JWK001 Injection"The Investigational New Drug (IND) application for the new drug has been successfully approved by the National Medical Products Administration (NMPA).
Source: CDE official website
JWK001The injection is the first AAV gene therapy new drug to adopt the "two-plasmid packaging system."Jinweike Biotechnology's self-developed two-plasmid suspensionHEK293 Cell Packaging Technology Significantly Enhances AAV Packaging Efficiency and Reduces Production Costs. The CMC process of Jinweike Biotechnology is stable and cost-effective, offering significant advantages during both clinical and commercial stages, greatly improving patient accessibility.On February 26, SANEGENEBIO announced that its self-developed siRNA drug SGB-9768 for the treatment of complement-related diseases has recently been approved by New Zealand's Medicines and Medical Devices Safety Authority (Medsafe) and the Health and Disability Ethics Committee (HDEC) to conduct Phase I clinical trials in New Zealand. This is the second siRNA drug developed by SANEGENEBIO to enter the clinical trial stage.
SGB-9768 is a siRNA-GalNAc conjugate targeting complement C3, delivered to liver cells using SANEGENEBIO's uniquely innovative next-generation LEAD™ GalNAc technology, inhibiting the synthesis of liver C3 via RNAi. The safety, efficacy, and stability of GalNAc-delivered siRNA drugs have been extensively validated by a large amount of data.This clinical study is a Phase I, randomized, double-blind, placebo-controlled, single-dose escalation trial. The primary objective is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SGB-9768 in healthy volunteers.
R&D Pipeline
Summary
Since the market launch of CAR-T therapies Kymriah, Yescarta, and gene therapy Zolgensma, their sales have grown significantly. CGT therapies remain a highly sought-after field, with numerous pharmaceutical companies vying to secure their position in the race.In addition to the above 19 CGT therapies, at the beginning of March this yearThree CGT therapies have been approved, namely GCK-01 Cell Injection, YOLT-201, and XMVA09 Injection. Additionally, in February, the IND applications for Xiangxue Life Sciences' TCR-T product and Junsai Bio's non-viral vector gene-modified TIL cell new drug were accepted, with both potentially entering the clinical stage. CHIGENOVO is also about to initiate a gene therapy clinical trial for ZM-02 in treating advanced retinitis pigmentosa.
Reference: Official websites of various companies
