11 CGT Therapies Receive IND Approvals, Marking a Turning Point for Gene Therapy Development
Epigenic
Gene Editing Technology Developer
NEUREGEN
Novel Gene Therapy Drug Developer
January 20, 2026
eMedClub News
According to the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）According to incomplete statistics from the official website and publicly available information, last week(January 12 - January 18), Over 30 Class 1 innovative drugs proposed for inclusion in the breakthrough therapy category / IND approved for clinical tacit consent / IND application accepted.
Among these, cutting-edge technologies represented by circular RNA, epigenetic editing, and in vivo direct reprogramming have collectively made breakthroughs, driving the expansion of gene therapy in China from rare diseases and ophthalmic conditions to major disease areas such as solid tumors and chronic metabolic disorders. The industry is now entering a new phase characterized by technological diversification and broader indications.
The following will introduce representative Class 1 innovative drugs approved for/under clinical application.
Epigenic:
Epigenetic Editing Therapy
Epigenic Therapeutics' intravenous antiviral drug EPI-003 injection has been approved for clinical trials, intended for the development of treatment for chronic hepatitis B. It uses an LNP delivery system to precisely deliver mRNA encoding epigenetic regulatory proteins and guide RNA targeting HBV genes to liver cells.Direct and Persistent Epigenetic Modifications of cccDNA and Integrated DNA, thereby suppressing the generation of all viral products at the transcription source.
Preclinical studies show that EPI-003 can significantly and continuously reduce hepatitis B surface antigen.（HBsAg）and HBV DNA levels, maintaining long-term efficacy after discontinuation, and demonstrating the potential to achieve functional or even complete cure of hepatitis B.
CUREMED:
IL-12 Circular mRNA
CUREMED's cmRNA1210, an intratumoral injection based on the circular mRNA platform for IL-12, has received IND approval and is expected to be developed for the treatment of advanced solid tumors.(such as melanoma, colorectal cancer, etc.)cmRNA1210 can encode single-chain human IL-12. After local injection at the tumor site, it is taken up by cells and expresses high concentrations of IL-12. IL-12 can enhance the cytotoxic effects of CD8 T cells and NK cells in the tumor microenvironment, activating the body's anti-tumor immunity.
Compared with traditional linear mRNA, the preparation process of circular mRNA simplifies the capping and tailing steps. CUREMED's independently developed novel cyclization framework, the Clean-PIE system, achieves precise and efficient cyclization without introducing exogenous sequences.Lower immunogenicity, higher expression efficiency and longer expression duration。
NEUREGEN:
In situ transdifferentiation tumor therapy
NEUREGEN's IND Application for NRG-103 Injection, an In Situ Transdifferentiation Cancer Therapy, Approved for InitiationTreatment for relapsed or progressiveHigh-grade glioma. It is transmitted through「In situ cell transdifferentiation + oncolytic virus + immune enhancementThe triple-mechanism design: Based on the selective replication and glioma cell-killing ability of a new generation of recombinant oncolytic viruses, and the enhancement of immune killing capacity through cytokines, the in-situ transdifferentiation technology is further utilized to convert residual/escaped tumor cells into non-proliferating cells. This multi-mechanistic approach aims to improve the therapeutic efficacy for high-grade gliomas and fundamentally address the challenges of tumor escape and recurrence.
Currently, NRG-103 has received the U.S. FDA IND approval and Orphan Drug Designation.
ViGeneron：
AAV Gene Therapy
ViGeneron's Subsidiary VG801 Receives IND Acceptance; Utilizes Proprietary Dual AAV Vector REVeRT, with Each Vector Carrying Half of the ABCA4 Coding Sequence, Producing Two Independent Pre-mRNA Molecules That Trans-Splice to Form a Complete mRNA, Which is Then Translated into a Functional Protein. VG801 Also Employs a Novel vgAAV Capsid Generated Based on ViGeneron’s Proprietary vgAAV Technology Platform, Designed for Broad Retinal Transduction.
Crystal Core Biotech:
Cyclic Peptide Targeted Radiopharmaceuticals
Crystal Core Biotech's Lutetium [177Lu]JH040182 Injection JH04 IND Application Approved for Development to Treat Fibroblast Activation Protein (FAP) Patients with advanced solid tumors who are positive.
January 20th (Tuesday) 19:00--20:30，MedCareer collaborates with ExCell Bio and Jiangsu Safelisi BiotechnologySpecial Feature Live Course: Precision Cancer Fighting Engine: The Synergistic Development of ICE Therapy and Bispecific/Multispecific Antibody Technologies.Welcome everyone, scan the code to participate.
JH04 is aCyclic Peptide-Targeted Radiopharmaceuticals, by targeting fibroblast activation protein overexpressed on the surface of tumor cells（FAP）"Positioning": Precisely deliver therapeutic radionuclides such as Lu-177 or Ac-225 to the interior of tumor lesions, achieving targeted destruction of tumor cells at the molecular level while causing minimal or no damage to surrounding normal tissues.
Compared with the fastest-progressing similar products globally, JH04 exhibits higher tumor enrichment and faster renal clearance, representing a qualitative improvement in both efficacy and safety.
For more IND progress of new drugs, please refer to the table.
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