Recently, the U.S. Food and Drug Administration (FDA) released重磅 information, announcing a "more flexible, risk-based, phased adaptation" regulatory approach for the Chemistry, Manufacturing, and Control (CMC) aspects in the development of Cell and Gene Therapy (CGT) products. For the CGT industry currently navigating through cycles, this is undoubtedly a significant boost.

On January 14, 2026, Langxin Biologics announced that the Phase III clinical trial (STELLAR study) of its ophthalmic gene therapy product LX102, led by Professor Xiaorong Li of Tianjin Medical University Eye Hospital, the leading clinical trial unit in China, successfully screened the first participant for the first Phase III gene therapy clinical trial in ophthalmology in China. This marks the official launch of a pivotal Phase III clinical study for the first gene therapy drug in China targeting neovascular age-related macular degeneration (nAMD).

Recently, Shanghai Youyin Pharmaceuticals (Euhearing Therapeutics), a pioneer in the field of global auditory disorder gene therapy, announced good news: its self-developed Class 1 innovative drug EHT102 Injection has officially obtained clinical trial (IND) approval through the "30-day Review Channel for Innovative Drug Clinical Trials" newly established by the National Medical Products Administration (NMPA) in September 2025. EHT102 is not only the first approved IND for ear gene therapy in China but also one of the first Class 1 innovative drugs to be successfully approved after the implementation of the new policy. The review cycle was significantly shortened from the standard 60 working days to within 30 working days from acceptance to approval. This milestone breakthrough not only demonstrates the regulatory authority's policy inclination towards innovative drugs with significant clinical value but also represents the highest recognition of Youyin Pharmaceuticals' scientific innovation capabilities, data completeness, and scientific rigor.

Recommended Reading：Congratulations on the IND approval of Youyin Pharma's EHT102 otology gene therapy project!

On January 12, 2026, at the 44th J.P. Morgan Healthcare Conference, Neurogene announced the latest progress and key milestone plans for 2026 regarding its investigational gene therapy, NGN-401.

NGN-401 is a gene therapy based on the AAV9 vector that uses EXACT™ technology to regulate MECP2 expression. The Embolden™ registrational trial commenced dosing in Q4 2025 and is expected to complete all enrollments by Q2 2026.

Recommended reading:NGN-401 registrational trial progresses smoothly, long-term follow-up data to be released in 2026

On January 14, 2026, a research team led by Professor Shi Jun from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences (CAMS), published a research paper titled "CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia" in the prestigious international medical journal, The New England Journal of Medicine (NEJM). The study utilized CD19-targeted CAR-T cell therapy (developed by Hekang Bio) to eliminate autoreactive B cells and plasma cells responsible for autoimmune hemolytic anemia (AIHA), achieving complete response in all 11 patients with refractory AIHA. It further elucidated the mechanisms of disease relapse. This marks the first global demonstration of the breakthrough efficacy of CD19-targeted CAR-T cell therapy in the field of AIHA, offering new hope for patients with relapsed/refractory AIHA.

Recommended Reading:China's First NEJM Paper in 2026: CAR-T Cell Therapy for Autoimmune Hemolytic Anemia

Recently, Judith Breuer's research team from University College London published an article in Nat Med.: Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy. The study conducted a case analysis on a type 1 spinal muscular atrophy (SMA1) patient who developed severe hepatitis after receiving onasemnogene abeparvovec (a gene therapy for SMA). Long-read and short-read metagenomic sequencing was performed on the patient’s liver tissue. Sequences of production plasmids with complex structures and recombination characteristics were identified. The vector genomes exhibited extensive fragmentation and concatemerization, along with numerous vector-human fusion junctions. Additionally, human β-herpesvirus 6B (HHV-6B) was detected in the liver. Further investigation involving more patients is required to determine whether the presence of production plasmid sequences or helper viruses contributes to the formation of these complex concatemeric DNA structures in the liver, and whether these factors are critical causes of hepatotoxicity following AAV gene therapy.

Recommended Reading:Nat Med.: Contaminant Plasmid Sequences and Fragmented Vector Genomes Found in Liver After AAV Gene Therapy

On January 14, 2026, Professor Li Zeng's team from Tufts University published a research article titled "A disease-severity-responsive nanoparticle enables potent ghrelin messenger RNA therapy in osteoarthritis" online in the journal Nature Nanotechnology. The study proposes a nano-delivery strategy with adaptive responsiveness to disease severity for precise mRNA treatment of osteoarthritis (OA).

The research team designed a negatively charged PLGA nanoparticle system based on the key pathological feature of progressive loss of glycosaminoglycans and reduced negative charge in the cartilage extracellular matrix during OA progression, introducing a novel concept called "matrix inverse targeting (MINT)." These nanoparticles selectively accumulate in areas of cartilage lesions with more severe degeneration, enabling enhanced delivery and expression as the disease worsens. In a mouse model of OA, MINT nanoparticles delivering ghrelin mRNA significantly reduced cartilage degeneration and subchondral bone abnormalities while effectively alleviating pain responses, demonstrating clear disease-modifying potential. This study provides a promising new strategy with translational potential for the precise application of non-viral RNA therapies in degenerative joint diseases.

2026On January 13, Zhang Xiaomin and Li Xiaorong from Tianjin Medical University published a research article titled "Small intracellular vesicles outperform small extracellular vesicles in uptake, drug delivery and retinal neuroprotection" in Nature Biomedical Engineering. The study shows that intracellular vesicles outperform extracellular vesicles in terms of uptake, drug delivery, and retinal neuroprotection.

In this study, researchers developed protocols for isolating intracellular vesicles from different cell types. Due to their small size, these isolated vesicles are referred to as small intracellular vesicles (sIVs). sIVs are smaller than small extracellular vesicles (sEVs) and feature simpler isolation methods, higher yields, unique molecular characteristics, and enhanced drug delivery capabilities. Their biological properties and potential therapeutic applications were also investigated. Small intracellular vesicles (sIVs) derived from umbilical cord mesenchymal stem cells (MSCs) more effectively alleviated retinal degeneration by reducing ER stress and providing neuroprotective factors. This study demonstrates that sIVs are a superior alternative to traditional bio-nanovesicles and can be utilized for clinical translation.