Innostellar Biotherapeutics Receives CDE Approval for LX101 Gene Therapy Clinical Trial in China for RPE65-Mediated Inherited Retinal Degeneration

April 19, 2022 – Shanghai Langsheng Biotechnology Co., Ltd., a subsidiary of Innostellar Biotherapeutics Co., Ltd., announced that the clinical trial application for its product LX101 injection to treat inherited retinal degeneration (IRD) patients with RPE65 biallelic mutations has been approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration.

RPE65Inherited Retinal Degenerations (IRD) caused by genetic mutations are a typical blinding rare disease. Patients develop symptoms during infancy or early childhood, usually experiencing severe, progressive retinal degeneration and deterioration of visual function, with almost all patients eventually progressing to complete blindness. In the past, there were no effective treatments. However, gene therapy, as a breakthrough technology, offers the possibility of fundamentally supplementing or repairing defective genes and restoring the normal biological function of healthy genes. According to existing observational data from related products abroad, gene therapy can help improve and maintain vision function in patients with this type of IRD over the long term.

Dr. Fenghua Wang, CEO of Innostellar Biotherapeutics"This is an important milestone since the company’s establishment. We would like to thank all the staff and collaborators at Innostellar Biotherapeutics for their hard work, as well as the trust and support from patients. Since 2021, when we collaborated with the National Clinical Research Center for Ocular Diseases to initiate China's first Investigator-Initiated Trial (IIT) clinical study on gene therapy for IRD, LX101 has been progressively demonstrating its therapeutic potential. We look forward to LX101 bringing more breakthroughs and maximum efficacy to patients, and we are even more hopeful that gene therapy will provide effective solutions in restoring healthy living for patients with other ocular diseases, genetic disorders, and chronic conditions."

LX101 Injection is a gene therapy independently developed by Innostellar Biotherapeutics using rAAV as a vector. The product works byRPE65Optimized design of gene coding sequences for efficient expression of human RPE65 protein, compensating for the protein deficiency caused by mutations in this gene. Intravitreal administration can effectively treat patients with inherited retinal degeneration (IRD) associated with biallelic RPE65 mutations. In previously conducted investigator-initiated clinical studies, LX101 demonstrated good safety and has shown efficacy in improving vision among multiple patients.

Innostellar Biotherapeutics Co., Ltd. is an innovative biotechnology company dedicated to the research and development of gene therapy products. Established in 2020, the company’s core team consists of scientists and professionals from globally renowned universities and laboratories, as well as senior technical experts and management personnel from top-tier global biopharmaceutical companies. Since its inception, the company has closely collaborated with clinical medical scientists from well-known hospitals in China, focusing on urgent needs in the treatment of genetic disorders and chronic diseases, and has accumulated a foundation for the development of multiple pipeline products and industrialization technologies.

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 technological transformation in gene therapy. It has established an independent research and development team and platform for ophthalmic gene therapy. The company possesses a world-leading serum-free suspension culture process platform and completed the construction of a nearly 5,000-square-meter facility for R&D and production in early 2021. After a year of rapid development, it has preliminarily built a full-process platform layout covering R&D, process, production, quality, regulatory submission, and clinical development.

The core members of the company have over 20 years of experience in innovative biopharmaceutical development and more than 10 years of experience in gene therapy research and development. They have led and participated in over 40 IND applications domestically and internationally, as well as the launch of three Class 1 new drug products. The international industrialization team has successfully scaled up a 500L AAV production process and possesses a serum-free suspension culture process platform that is currently leading internationally. In terms of external cooperation, the company focuses on clinical needs and collaborates with multiple hospitals and research institutions worldwide to advance the application and clinical research of gene therapy in hereditary retinal diseases, muscular disorders, and neurological conditions.