Twelve Domestic CGT Therapies Receive IND Approval in China in July 2024

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Cell and Gene Therapy (CGT) has become one of the most promising cutting-edge pharmaceutical fields globally.The first half of 2024,About 70 CGT therapy INDs have been approved in China. In the second half of the year,R&D in the CGT FieldProgress Reports Frequently,According to incomplete statistics, in July 2024, 12 more CGT therapies were approved for IND. The editor has compiled the following list:

On July 5, the innovative biological product MVR-T3011 herpesvirus injection, developed by Immvira Pharmaceuticals, received clinical trial approval from the National Medical Products Administration (NMPA) for intraperitoneal administration to treat advanced solid tumors.

MVR-T3011 is an innovative triple-combination oncolytic herpes virus product independently developed by ImmunoViro Therapeutics. Based on a completely new design of the wild-type HSV-I herpes virus backbone, it ensures robust replication capability of the virus within tumor cells while suppressing its replication in normal cells, achieving optimal attenuation. In addition, MVR-T3011 carries two cutting-edge and well-validated exogenous immune-modulating genes, PD-1 antibody and IL-12, to enhance the immune response in the tumor microenvironment. This enables the virus to lyse tumor cells while exerting dual therapeutic effects of oncolysis and immunotherapy.

On July 9, 2024, SANEGENEBIO announced the co-development with Innovent Bio of a small nucleic acid for the treatment of hypertension.(siRNA) Drug SGB-3908 InjectionThe clinical trial application has received the tacit approval from the CDE for the treatment of hypertension.This is the third clinical trial application in China by SANEGENEBIO, following the SGB-3403 Injection and SGB-9768 Injection.The Third siRNA Drug。

In December 2023, SANEGENEBIO and Innovent Bio announced a strategic collaboration agreement to co-develop the siRNA candidate drug SGB-3908 for the treatment of hypertension.According to the terms of the agreement, Innovent Bio has obtained an exclusive option and can pay the exercise fee in the future to acquire the exclusive rights for the development, production, and commercialization of SGB-3908 across different regions globally. After Innovent Bio exercises the option, SANEGENEBIO will also be entitled to subsequent R&D milestone payments and sales milestone payments, as well as tiered royalties based on net sales post-commercialization. The company has not disclosed the financial details of the collaboration.

On July 15, according to the CDE official website, "CEL001 Injection" developed by Guangzhou Xiling Biotechnology Co., Ltd. ("Xiling Bio" for short) received clinical trial implied permission, with the indication for advanced solid tumors.This is the first non-gene-edited allogeneic NK cell candidate product in China to have applied for an IND and received approval from the CDE.

CEL001 Injection, independently developed by SinoBio, is derived from the peripheral blood of healthy individuals. Based on in vitro culture technology and through steps such as isolation, activation, and proliferation, it is a universal off-the-shelf NK cell candidate product with high expression of activating receptors, high levels of granzyme and perforin expression, and both cytokine secretion function and cytotoxic effects.

On July 18, 2024, according to the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration, the Investigational New Drug (IND) application for AFN0328 Injection, a Class 1 new drug jointly developed by Hefei Alphana Biotechnology Co., Ltd. ("Alphana Bio") and Anhui Ankang Biological Engineering (Group) Co., Ltd. ("Ankang Bio"), was approved, becomingThe Second in ChinaHPV-related tumor mRNA therapeutic vaccine approved for clinical trials.

AFN0328 Injection is an mRNA drug for cancer treatment, jointly developed by the Afana Bio team and the Anke Bio team.AFN0328 adopts an adjuvant system with independent intellectual property rights, demonstrating significant advantages over existing systems on the market. In addition, this injection utilizes innovative antigen design, effectively presenting antigens in primates. After process upgrades and further formulation innovations, it has resolved challenges related to cold chain transportation and long-term storage. Notably, the sequence design of AFN0328 utilized the MiQro RNA drug design platform jointly released by SANEGENEBIO and its collaborators.

On May 8, 2024, an mRNA drug also named AFN0328 had its application formally accepted by the CDE (Acceptance No.: CXSL2400288). The applied indication is a First-in-class therapeutic mRNA vaccine targeting precancerous lesions. This mRNA-based strategy can precisely modulate the immune system, activating the body’s inherent cancer prevention mechanisms to block cancer progression during the early to middle stages of the disease. Intervening at the precancerous lesion stage is generally easier and more effective than treating after cancer has already developed, providing significant contributions to the field of cancer prevention and early treatment. It is reported that the project successfully held the Phase I/II clinical trial investigator meeting on July 5 at the First Affiliated Hospital of Anhui Medical University.

On July 22, RuiZheng Gene announced its independently developed treatment for transthyretin amyloidosis (ATTR).ART001, as the first non-viral vector in vivo gene editing drug in China to enter human clinical research (IIT), received approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration on July 19, 2024.Approval for Clinical TrialsTest。

ART001 Injection, developed by RuiZheng Gene, delivers CRISPR genome editing components to the liver via lipid nanoparticles (LNP) to edit the TTR gene, thereby blocking the expression of TTR protein and preventing the abnormal deposition of amyloid substances at the source.

On July 23, 2024, according to the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA), the "TAL-T Cell Injection" applied for by Guangzhou Fanen Biotechnology Co., Ltd. (hereinafter referred to as "Fanen Bio" or "the Company") has obtained clinical trial tacit permission. The indication is advanced malignant solid tumors (Acceptance No. CXSL2400285).

The cell injection approved for IND this time is the world's first TAL-T cell therapy, developed by FanEn Bio using its proprietary core technology to prepare high-quality autologous anti-tumor T cells from tumor-associated lymph nodes. This therapy successfully addresses the challenges faced by existing autologous anti-tumor T cell therapies like TIL, such as low preparation success rates and suitability for only a small subset of patients, enabling the successful application of autologous anti-tumor T cell therapy to the majority of solid tumor patients. TAL-T cell therapy offers advantages such as broad indications, no genetic modification, short preparation cycles, and excellent therapeutic effects. It is currently undergoing IIT clinical research, demonstrating favorable safety and significant efficacy.

Among the 975 biopharmaceutical new drug applications accepted in 2023, a total of 167 CGT products were accepted for IND. Among them, 48 applications were for stem cell products, 73 for immune cell products, and 45 for gene therapy products.In the first half of 2024, 70 CGT drugs have been approved for clinical trials. It is believed that in the future, a large number of cell and gene therapy projects under research will begin to enter the clinical stage in a concentrated manner.

References: Announcements from various companies, CDE official website

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