Breakthrough Gene Therapies Gain Market Approval in China—Is a Wave of Commercialization Imminent?

From Naked Plasmids to AAV Vector-Delivered Therapeutics: China’s Gene Therapy Sector Is Entering a Period of Intensive Harvest

On May 28, Northland Sedominji(Huasuo Ling)Approved for market launch, becoming the first domestic therapy for severe lower limb ischemia（CLI）of naked plasmid gene drugs. Previously, Belief BioMed's bope...(Xinjiuning)Approved in April 2025, it became the first AAV gene therapy drug in China for the treatment of hemophilia B.

In the field of gene editing, although no products have been approved for market launch, multiple pipelines(Including in vivo and ex vivo gene editing drugs)Has entered the critical clinical stage,The first approved drug will soon be launched.

Representative Gene Therapy Drugs Entering Phase III/Pivotal Clinical Stage(Listed in no particular order)。

Naked plasmids are circular double-stranded DNA plasmids carrying therapeutic gene expression cassettes. They are commonly used to deliver therapeutic genes to target cells via local injection, leveraging the host cell’s own expression machinery to sustainably produce therapeutic proteins. As a non-viral delivery system, their advantages include simple manufacturing processes, lower costs, and the absence of virus-related safety concerns.

Currently, Sedomingji is the only approved naked plasmid gene therapy drug marketed in China. The drug delivers hepatocyte growth factor via intramuscular injection into the leg muscles.（HGF）Naked plasmid DNA was delivered to the ischemic site, where it transfected skeletal muscle cells to express HGF, thereby promoting local angiogenesis and improving limb blood supply.

Phase III HOPE-CLTI-2 Study Shows(Treatment for 180 days), the complete ulcer healing rate in the Cedomingji group reached43.5%(Placebo group 18.5%), the amputation rate was only1.9%(Placebo group 8.8%), the risk of amputation or death was reduced compared with placebo87%（HR=0.13，P=0.0024）。

Furthermore, the Phase III clinical trials for Sedomingji in the indication of rest pain have been completed, and development is underway for a new indication in intermittent claudication. In 2024, Humanwell Healthcare Group Co. Ltd. submitted an application for a candidate drug with the same target and class, namely Recombinant Plasmid-Hepatocyte Growth Factor Injection.（pUDK-HGF）marketing application, which was voluntarily withdrawn in December 2025, with the statement that it would be resubmitted after the data were perfected.

AAV gene therapy is currently one of the most commercially mature and widely clinically applied types of gene therapy. Leveraging its favorable safety profile, low immunogenicity, and long-term gene expression capabilities, it is suitable for indications requiring durable correction of genetic defects, such as rare diseases, hemophilia, ophthalmic disorders, and neurological diseases.

In addition to Belief BioMed’s first approved drug, at least eight AAV gene therapy drugs in China have entered Phase III clinical trials. Companies such as Zhongyin Technology are expected to submit marketing applications this year, vying to launch the second AAV drug in the domestic market.

In terms of indication distribution, the majority of Phase III clinical pipelines are focused on the ophthalmology field, including Langxin Biologics LX101/LX102, targeting inherited retinal dystrophies and neovascular age-related macular degeneration, respectively; Fangtuo Biologics' FT-001, targetingHereditary Retinal DystrophyMalnutrition；Zhongyin TechnologyZVS-101e, forCrystalline Retinal Degeneration; Tianze Yuntai VGR-R01, for crystalline retinal dystrophy; Nuofusi Biologics NR082, for Leber hereditary optic neuropathy, etc.

Jinlan Gene and Huayi Lejian have avoided the highly competitive ophthalmology sector, opting instead for differentiated strategic layouts. The former’s GC101 is the first intrathecal AAV gene therapy candidate drug for spinal muscular atrophy (SMA) in China, while the latter’s GS1191-0445 targets hemophilia A.

In addition to AAV delivery vectors, the active ingredient of Weiyuan Likang’s WG1025 is a replication-defective HSV-1 vector carrying the human COL7A1 gene. It completed treatment for dystrophic epidermolysis bullosa in January 2026.（DEB）a randomized, double-blind, placebo-controlled confirmatory clinical study.

Gene editing therapies leverage tools such as the CRISPR-Cas system to precisely modify the genome, fundamentally correcting disease-causing mutations, with several candidates having advanced into pivotal clinical stages.

Ruifeng Bio's Lead Pipeline RM-001(HBG Gene-Modified Autologous CD34+ Hematopoietic Stem Cell Injection)It is an ex vivo gene-editing candidate drug for β-thalassemia, targeting the HBG gene. As of November 2025, it has completed confirmatory clinical trials and is in the follow-up phase.

BRL-101, developed by Bangyao Biotechnology, is the world’s first gene therapy candidate utilizing gene-editing technology to treat β0/β0-type severe beta-thalassemia and is currently in the pivotal Phase II clinical trial stage for regulatory approval. This therapy is built upon a proprietary hematopoietic stem cell platform with independent intellectual property rights.（ModiHSC）Development: The use of electroporation to deliver target genes fundamentally eliminates the potential tumorigenic risk associated with viral integration.

In vivo gene editing therapies are also progressing steadily. Yaotang Biopharma’s in vivo gene editing candidate drug YOLT-203, delivered via mRNA-LNP, targets the HAO1 gene. In November 2025, it received FDA approval to initiate the world’s first clinical trial for primary hyperoxaluria type 1.（PH1）Confirmatory Clinical Trial of In Vivo Gene Editing Therapy.

The approval and market launch of two “first-in-class” drugs mark China’s transition in gene therapy from research and development to commercialization. Yet this is only the beginning. As more Phase III/key clinical pipelines advance, and as commercial pricing, medical insurance reimbursement, and manufacturing processes gradually mature, China’s gene therapy sector will accelerate toward broad clinical accessibility, ushering in a true era of industrialization.

Responsible Editor | Ju

Proofreader: Ju

References:

1. CDE and Official Materials from Various Companies

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