Anlong Bio Receives IND Approval for AL-001 Gene Therapy in Diabetic Retinopathy

DR is the leading cause of blindness among working-age populations. China has the highest number of people with diabetes globally. The latest diabetes map from the National Center for Chronic and Noncommunicable Disease Control and Prevention shows that, as of 2023, China had 233 million diabetes patients, with over 34% having DR. Due to the lack of a first-line treatment across all stages, blindness and low vision caused by DR have become a major public health challenge.

As the first product in China and the second globally to deliver gene therapy via the suprachoroidal route, AL-001 has set a record as the "world's first to express aflibercept through this pathway." Studies both in China and internationally have confirmed that the suprachoroidal drug delivery method allows the drug to circulate around the entire eye, overcoming the limitations of the macula, making it more suitable for the pathological characteristics of DR, which affects the full-layer structure of the retina. It is also currently the safest method of administering AAV vector drugs — which is particularly important for DR patients. In 2021, a similar product by the U.S.-based Adverum company using intravitreal injection led to clinical accidents in DR&DME trials and ultimately failed, severely impacting the application of AAV vector drugs in chronic ocular disease treatments. Later, the U.S.-based RegenxBio company adopted suprachoroidal administration, demonstrating superior safety and efficacy in Phase II clinical trials for DR, becoming a key outcome that facilitated a $1.75 billion collaboration with AbbVie. Suprachoroidal administration is crucial for gene therapy drugs entering DR clinical trials.

Professor Youxin Chen from Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, pointed out:Gene therapy is not a replacement for protein therapy but an important complement. In the future, after patients are diagnosed, they may first receive protein therapy, and once its efficacy is confirmed, gene therapy could replace the subsequent frequent injections. With a price similar to protein therapy, it can effectively avoid the physical trauma and economic pressure caused by continuous drug administration. Safe and effective gene therapy drugs are expected to become the top choice for both doctors and patients. On one hand, it significantly reduces the burden on patients; on the other hand, it saves enormous medical costs for national health insurance in China.

Dr. Chunlin Zhao, founder and CEO of Anlong, stated:The simultaneous advancement of DR and wAMD indications reflects Anlong's R&D strategy guided by clinical needs. We look forward to providing patients with ocular diseases a "once-and-for-all" treatment option through gene therapy technology, accelerating the clinical development process, and promoting the commercialization of products.

Anlong is a national high-tech and specialized enterprise focusing on gene therapy and nucleic acid drug research and development. It is committed to developing innovative drugs based on cutting-edge technologies such as siRNA (small interfering RNA) and AAV (adeno-associated virus). The company focuses on fields with huge market potential, prioritizing the treatment of chronic diseases, metabolic diseases, and neurological disorders, while developing breakthrough therapies with independent intellectual property rights. Relying on two major technology platforms—AAV gene therapy and siRNA small nucleic acid drugs—Anlong is accelerating the development of multiple innovative drugs. Through close cooperation with top domestic and international scientific research institutions and enterprises, the company drives innovation-led growth, aiming to become a leader in China's gene drug sector and provide patients with affordable and innovative treatment solutions.