32 CGT Therapies Receive IND Approval in China During Q1 2024

Apr 01, 2024 14:07 CST Updated 14:07

CUNDE

Tumor Immunotherapy Technology and Drug Developer

SANEGENEBIO

Small Nucleic Acid Drug Developer

Disclaimer: Due to limited expertise, errors are inevitable, and some information may not be the most up-to-date. Feel free to point out any issues in the comments. This article is only an introduction to medical and health-related drugs, not a recommendation of treatment plans (if involved); it does not constitute any investment advice.

Cell and Gene Therapy (CGT) is a new generation of precision therapy following small molecule and large molecule targeted therapies.According to incomplete statistics, from January to March 2024, 32 CGT therapies have been approved for IND, with CAR-T therapy drugs becoming the mainstream among the approved CGT drugs. The approved drugs include 22 cell products and 10 gene therapy products. The following is a summary compiled by the editor:

CUNDE

On March 21, the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) announced that CUD005, a macrophage injection independently developed by Sichuan Cunde Therapeutics Co., Ltd. ("CUNDE"), a company in which Lai Mei Pharmaceuticals holds shares, received approval for its Investigational New Drug (IND) application to treat mid-to-late stage liver cirrhosis.

CUD005 Injection is a treatment based on the patient's own immune cells, which are differentiated into mature and stable individualized anti-fibrotic macrophages. Compared with traditional small-molecule drugs, CUD005 Injection can effectively degrade the extracellular matrix by secreting various matrix metalloproteinases (MMPs), reducing liver scar accumulation. At the same time, it releases anti-inflammatory factors (such as IL-1RN) to suppress inflammatory responses, improve the liver’s inflammatory microenvironment, and slow disease progression. Additionally, CUD005 Injection demonstrates strong phagocytic ability for apoptotic cells, promoting the restoration of normal liver structure. Currently, there have been no reports of other candidate macrophage therapies for decompensated cirrhosis being approved for clinical use.

Shandong Qilu Cell Therapy Engineering Technology Co.,LTD

On March 11, the human umbilical cord mesenchymal stem cell injection (code name YFQLXB-UC01), developed over four years by Dr. Ma Heran's team at Shandong Qilu Cell Therapy Engineering Technology Co., LTD under Yinfeng Biotechnology Group, received the "Drug Clinical Trial Approval Notice" issued by the National Medical Products Administration, with indications for decompensated liver cirrhosis. This marks another milestone for Yinfeng Biotechnology Group in the field of liver cirrhosis following two IIT studies registered with the National Health Commission, and it is also the first IND product for this indication within Shandong Province.

YFQLXB-UC01 Injection is a cryopreserved cell preparation independently developed by Qilu Cell Company of Yinfeng Biotechnology Group. The mesenchymal stem cells are derived from Wharton's Jelly of the umbilical cord tissue of healthy newborns. After isolation and expansion in vitro, the stem cells are mixed with clinical-grade cryoprotectant to produce the stem cell preparation. Unlike traditional fresh preparations, this formulation is an "off-the-shelf" product that can be used for intravenous infusion without washing after revival.

SANEGENEBIO

On February 26, SANEGENEBIO announced that its self-developed siRNA drug SGB-9768 for the treatment of complement-related diseases had 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 from 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 hepatic C3 through 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

GOLDENWEKE BIO

On February 22, Genevector BioIndependently Developed for Neovascular Age-Related Macular Degeneration(nAMD) Gene Therapy Product"JWK001 Injection"The IND application for the new drug has been successfully approved by the National Medical Products Administration (NMPA).

Image Source: CDE Official Website

JWK001 Injection is the first AAV gene therapy new drug to adopt a "two-plasmid packaging system." The two-plasmid suspension HEK293 cell packaging technology independently developed by Jinwei Ke significantly improves AAV packaging efficiency and reduces production costs. Jinwei Ke’s CMC process is stable and cost-effective, offering significant advantages during both the clinical and commercialization stages, greatly enhancing patient accessibility.

Eternity Bio

On February 18, YonTech Bio-B (06978.HK) announced that its aT19 injection has successfully obtained clinical trial approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA).

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 aT19 and CAR-T-19 injection for the treatment of CD19-positive relapsed/refractory B-cell acute lymphoblastic leukemia in patients aged 25 years or younger. The proposed indication is for relapsed/refractory B-cell acute lymphoblastic leukemia in patients aged 25 years or younger who have achieved clinical benefit from CD19-targeted CAR-T cell therapy, aiming to enhance treatment efficacy and reduce recurrence. This trial is scheduled to commence within 2024.

Easymore

On February 7, ImmunoCell 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), intended for the treatment of advanced digestive system tumors with positive EpCAM expression, including but not limited to advanced gastric cancer (GC)/gastroesophageal junction adenocarcinoma (GEJ).

IMC001 BecomesThe World's First CAR-T Product Targeting EpCAM Approved for IND; IMC001 has demonstrated good safety and efficacy in IIT studies.

On February 23, ImmunoCell Bio announced that its self-developed autologous CAR-T cell injection product (IMC001) targeting EpCAM, after receiving clinical trial approval from China's CDE, has recently been granted clinical research authorization by the U.S. FDA for the treatment of EpCAM-positive advanced digestive system tumors, including but not limited to advanced gastric cancer (GC)/gastroesophageal junction adenocarcinoma (GEJ).

ORIGIBIO

On January 29, 2024, Original Biotech 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 Yuanqi Bio using its proprietary technology platforms. The design and development of this product incorporate Yuanqi Bio'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, Yuanqi Bio is about to initiate clinical development of OriCAR-017 in the United States.

R&D Pipeline

ZHONGJI INTELLIGENT PHARMACEUTICALS

On January 26, 2024, the Investigational New Drug (IND) application for GMCN-508B, a gene therapy for β-thalassemia submitted by Zhongji Zhiyao, 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 Zhongji Zhiyao, an emerging company focusing on lentiviral vector and hematopoietic stem cell therapy, signifying its successful transformation from "scientists" to "pharmaceutical professionals."

Summary

Among the 975 biopharmaceutical new drug applications accepted in 2023, a total of 167 CGT products received IND acceptance. Among these, there were 48 stem cell product applications, 73 immune cell product applications, and 45 gene therapy product applications.

2024 will also be a breakout year for CGT therapies, with many milestone events in the CGT field occurring just in the first quarter, such asFFDA Approves a Lentiviral Vector Gene DrugLenmeldyLaunched at a price of $4.25 million, currently the world's most expensive drug; Pfizer'sGene therapy Beqvez has also been approved for marketing;Huadong Medicine and Carsgen Biotech's BCMA CAR-T Collaborative Product, Sekize®, Approved for Marketing in China;World's First TIL TherapyAmtagviApproved for marketing, priced at $515,000,A New Era for Solid Tumor Treatment.CGT Has Huge Future Development Space, BelieveCGT can provide new options for more difficult-to-treat diseases in the future.

References: Official websites of various companies