GeneTherapy Biotech Jinlan Gene Submits IND for GC310, an AAV-Based Gene Therapy for Wilson Disease

Nov 18, 2024 20:32 CST Updated 20:32

Genecradle

Gene Therapy Drug Developer

Source: "BJGC Genecradle" WeChat Official Account

Recently, GC310 Adeno-Associated Virus Injection, a new product independently developed by Beijing Genecradle Therapeutics Co., Ltd. ("Genecradle"), has been accepted for clinical trial registration review by the National Medical Products Administration. The drug is registered as a Class 1 therapeutic biological product, indicated for Wilson’s disease. This marks the fourth gene therapy drug product from Genecradle to be submitted for clinical trial following GC101, GC301, and GC304.

GC310 Adeno-Associated Virus Injection

GC310 AAV Injection is an AAV gene therapy drug independently developed by Genecradle for the treatment of Wilson’s disease. Non-clinical studies have demonstrated good drug safety and significant efficacy. A single treatment enables the target tissue to express the miniATP7B copper transporter with biological function, restoring copper metabolism and increasing ceruloplasmin levels, which is expected to improve the condition of the Wilson’s disease community in the long term.

Hepatolenticular Degeneration

Also known as Wilson's disease (Wilson Disease, WD), it is an autosomal recessive genetic disorder caused by mutations in the ATP7B gene. Functional defects in the ATP7B gene lead to impaired copper metabolism, resulting in the abnormal accumulation of toxic free copper ions in the body, primarily depositing in the liver, central nervous system, kidneys, and cornea, causing a series of progressively worsening pathological changes such as cirrhosis, neuropsychiatric disorders, renal dysfunction, and the characteristic formation of corneal K-F rings.

Globally, the prevalence of WD is approximately 1 in 30,000 to 100,000 people, while the carrier rate of the disease-causing gene is about 1/90. The age of onset for this condition varies widely but is mainly concentrated in children and adolescents, with a peak incidence between the ages of 5 and 35. Notably, epidemiological studies suggest that the incidence and carrier rates of WD may be higher in Asian regions such as China compared to Western countries, with some data indicating that the incidence in China could be close to 1 in 10,000.

In terms of treatment, hepatolenticular degeneration faces significant clinical challenges. The current standard treatment regimen requires patients to undergo frequent drug interventions for the long term or even lifelong after diagnosis. However, these treatments cannot fully restore the normal metabolic balance of copper ions within tissue cells. As the disease progresses, if not effectively controlled, it will inevitably worsen to the middle or late stages, posing a severe threat to the patient’s life. Therefore, it is particularly urgent to explore more efficient treatment strategies that can fundamentally improve abnormal copper metabolism.

Genecradle

Genecradle is a national high-tech enterprise with the core business of developing gene therapy drugs mediated by AAV vector delivery technology. It aims to advance China’s rare disease gene drugs from basic research to clinical and market applications, benefiting patients and their families. The company focuses on the development of gene therapy drugs for hereditary neuromuscular diseases, inherited metabolic disorders, lysosomal diseases, and ophthalmic diseases. By promoting the research, development, and clinical application of rare disease gene drugs, it seeks to deepen the understanding of life and health, transitioning gene therapy technologies and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.