Continuous Breakthroughs in R&D: Chinese Biotechs Score Frequent Victories in Gene Therapy

Chinese biotech firms are racking up a string of breakthroughs in gene therapy — from clinical validation and platform innovation to a fundamental expansion of therapeutic paradigms.

The global gene therapy market is surging. According to Business Research Insights, the market stood at USD 9.03 billion in 2024 and is projected to reach USD 64.64 billion by 2033, growing at a compound annual growth rate of 27.6%. Against that backdrop, pharmaceutical companies worldwide are pouring resources into gene therapy — and Chinese firms are now beginning to reap returns.

In recent weeks, a wave of milestones from China's biotech sector has underscored the sector's momentum, spanning clinical efficacy validation, next-generation platform technologies, and regulatory landmarks.

Zhongmou Therapeutics Advances Optogenetic Gene Therapy

Wuhan Hiteck Biological Pharma Co., Ltd. announced recently that its associate company Zhongmou Therapeutics has received clinical trial approval from China's National Medical Products Administration (NMPA) for its ZM-02 ophthalmic injection, a next-generation optogenetic gene therapy targeting advanced retinitis pigmentosa (RP).

ZM-02 is designed to treat advanced retinal degenerative diseases. Preclinical studies demonstrated stable protein expression in RP mouse models, along with notable photosensitivity and visual function recovery. In the ongoing human MOON clinical trial, the therapy showed a favorable safety profile and significant improvements in both vision and quality of life for patients with advanced RP.

In October 2024, ZM-02 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) — a significant regulatory milestone that underscores the therapy's potential in a rare disease with limited treatment options.

Fangtuo Biologics Wins FDA RMAT Designation for AAV Gene Therapy

On July 29, Fangtuo Biologics announced that the U.S. FDA has granted its lead candidate FT-003 a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of neovascular age-related macular degeneration (nAMD).

FT-003 is an intravitreal AAV gene therapy product that delivers a codon-optimized aflibercept encoding sequence. The goal: achieving long-term, sustained intraocular anti-VEGF expression from a single administration — dramatically reducing the injection burden on patients while maintaining visual benefits.

Phase II clinical results showed that FT-003 not only improved visual acuity, eliminated retinal edema, and improved retinal structure in nAMD patients, but also substantially reduced the need for repeated anti-VEGF treatments. The therapy demonstrated good safety and tolerability.

Kanghong Pharmaceutical Pushes Gene Therapy into Cardiology

On July 23, Chengdu Kanghong Pharmaceutical Group Co., Ltd. disclosed that its subsidiary Chengdu Hongji Biotechnology Co., Ltd. has received NMPA approval to initiate clinical trials for KHN921 injection, an AAV gene therapy product targeting hypertrophic cardiomyopathy caused by MYBPC3 mutations.

The program marks a notable expansion of gene therapy beyond ophthalmology and rare blood disorders into cardiovascular disease. In April 2026, the FDA had already granted permission for KHN921 to proceed with clinical trials in the United States, signaling international recognition of the program's potential.

Vertex's Casgevy Expands to Younger Patients

In July, Vertex Pharmaceuticals announced that the FDA has formally approved a supplemental label expansion for its gene therapy Casgevy. The approval lowers the minimum age to 2 years and above for patients with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOC), as well as for patients with transfusion-dependent beta thalassemia (TDT).

Casgevy is a CRISPR/Cas9-based ex vivo gene editing therapy. The procedure involves extracting a patient's own hematopoietic stem cells, genetically modifying them in the laboratory, and returning the repaired cells to the patient's body. Before this latest expansion, Casgevy had already secured a triple regulatory fast-track — orphan drug designation, RMAT, and Fast Track status.

From Policy-Driven to Capability-Driven

Taken together, these developments paint a clear picture: China's gene therapy sector is undergoing a fundamental shift — from policy-driven growth to capability-driven competitiveness. As domestic firms continue to break through in foundational technology innovation and industrialization capacity, their standing in the global gene therapy arena is set to rise. In certain subfields, Chinese companies may soon achieve parity with — or even lead — their international peers.

Disclaimer: The information contained in this article does not constitute investment advice in any circumstances.