Gene Therapy Achieves Major Breakthrough: Innostellar Biotherapeutics Announces Promising LX102 Clinical Data That May Redefine Treatment Paradigm for Age-Related Macular Degeneration

Recently, Innostellar Biotherapeutics was invited to attend the 2025 Annual Meeting of Macula Society and the Angiogenesis, Exudation, and Degeneration Conference in the United States, where it released the latest clinical trial results of LX102, a gene therapy for neovascular age-related macular degeneration (nAMD) independently developed by the company. Professor Xiaodong Sun from the National Clinical Research Center for Ocular Diseases and Shanghai First People's Hospital, as the principal investigator, provided a detailed introduction of the latest data from the 12-month follow-up of the Phase I clinical trial in China and the 24-month follow-up of the investigator-initiated trial (IIT). As the first registrational clinical trial in China for a gene therapy treating AMD, the data released this time demonstrated the excellent safety and outstanding efficacy of LX102, providing strong evidence for the application of gene therapy in major diseases like AMD.

All patients in the Phase I study of LX102 have completed at least one year of observation, and none of the 9 subjects who received medium to high doses required supplemental anti-VEGF drug treatment.The first subject of LX102IIT has completed a 30-month follow-up with sustained and stable improvement in vision. A total of six patients have completed at least 24 months of follow-up without the need for any additional treatment during this period.

These patients involved in the study had an average age of 71 years and received 8 to 9 annualized anti-VEGF treatments before receiving LX102. The visual improvement brought by LX102 is similar to that of existing anti-VEGF antibody or fusion protein drugs currently on the market, and the lesion activity in all studied eyes has been stably controlled.

Gene Therapy for AMD is One of the Hot Topics at This Conference. Two products from the United States also reported relevant clinical trial results. The Phase II clinical study results of 4D-150 showed that 43% of subjects in the treatment dose group required anti-VEGF supplemental treatment after 52 weeks post-administration; the Phase II clinical study results of ADVM-022 showed that 39% of subjects needed anti-VEGF supplemental treatment after 52 weeks post-administration.

Currently, more than 60 patients have received LX102 treatment, compared to similar products globally.，LX102 truly achieves "one administration, long-lasting effectiveness," realizing the goal of "zero rescue" at the target dose while requiring a lower effective dose, further demonstrating the superior efficacy trend of LX102.The product is expected to enter Phase III clinical trials by the end of this year.

The experts at the meeting conducted an in-depth discussion on the above research results. Compared with existing long-acting treatment methods, gene therapy can achieve long-term stable control of the disease with a single administration.(Continuous expression or even lifelong), accumulating increasing data in terms of safety and efficacy, it is regarded as a promising solution to address the issue of long-term repeated invasive treatments for AMD patients. Particularly, LX102 adopts subretinal administration, eliminating the need for prophylactic use of corticosteroids, and no drug-related immune-inflammatory reactions have been observed, demonstrating significant safety advantages in the treatment of common eye diseases such as AMD. Moreover, through molecular design optimization combined with localized subretinal delivery, LX102 achieves enhanced specific expression in the outer retinal lesion areas, offering potential new therapeutic options for refractory PCV subtype patients in the future. With the accumulation of more clinical data, LX102 is expected to become a revolutionary therapy in nAMD treatment.

About Macula Society Annual Meeting

The Macula Society meeting, hosted by the American Academy of Ophthalmology, aims to promote research and development in the field of ophthalmology. It is an important academic conference in the international ophthalmology community, especially in the area of retinal diseases, gathering global experts and researchers to share the latest research findings and clinical experiences.

About LX102 Injection

LX102 Injection, an nAMD gene therapy independently developed and manufactured by Innostellar Biotherapeutics, has a clear mechanism of action and demonstrated efficacy.This product uses an AAV vector to deliver DNA expressing an anti-VEGF fusion protein into retinal cells, thereby maintaining long-term expression of the anti-VEGF protein in the patient's eye.In the investigator-initiated clinical studies conducted previously, LX102 demonstrated good tolerability and has shown long-term efficacy in vision improvement in multiple patients.LX102 was granted clinical approval by China's CDE on December 23, 2022.On December 22, 2023, it received clinical approval from the U.S. FDA. 

About Innostellar Biotherapeutics

Innostellar Biotherapeutics Co., Ltd. is committed to the research, development, and manufacturing of innovative gene therapy drugs.Founded in 2020, the company focuses on the urgent needs in the treatment of genetic and chronic diseases, establishing a foundation for the development of multi-pipeline products.

The company focuses on ocular disease products as the breakthrough point for gene therapy, developing safe and precise gene replacement and gene editing technologies to achieve clinical applications of gene therapy. It has established a research and development team and platform production technology for gene therapy across multiple disease systems.Two products have officially received IND approval, both demonstrating good safety and efficacy trends in current clinical trials.LX101, used for treating RPE65 mutation-associated inherited retinal dystrophy (IRD), is the first IRD gene therapy product in China to receive clinical trial approval, with over 1 year of follow-up completed for 30 patients.The company focuses on the field of gene therapy for hereditary and chronic eye diseases, with a foundation in the development of multiple pipeline products and industrialization technologies. It closely collaborates with well-known clinical medical scientists both domestically and internationally to enhance innovation capabilities, developing gene therapy products with greater efficiency and higher quality, ultimately for clinical application.LX102 is Innostellar Biotherapeutics' representative product in the field of neovascular age-related macular degeneration (nAMD) and is currently undergoing clinical trials in multiple countries worldwide.