On February 6, 2025, Genecradle Therapeutics' independently developed gene therapy drug, GC310 Adeno-Associated Virus Injection ("GC310 Injection"), received the tacit approval for registration of clinical trials from the National Medical Products Administration (NMPA) (Application Numbers: CXSL2400774, CXSL2400775), with the indication of hepatolenticular degeneration. GC310 Injection is the fourth Class 1 new gene therapy drug from Genecradle Therapeutics to receive IND approval.
The clinical trial application for GC310 Injection was submitted on November 14, 2024, and was approved on February 6, 2025, after a rigorous review process. This efficient approval process not only reflects the NMPA's support for innovative drugs but also highlights Genecradle Therapeutics' R&D strength in the field of gene therapy.
Genecradle Therapeutics' IND Approvals at a Glance
Image Source: Screenshot from the official CDE website
Hepatolenticular degeneration, also known as Wilson Disease (WD), is an autosomal recessive inherited disorder caused by mutations in the ATP7B gene. The functional deficiency of the ATP7B gene leads to copper metabolism disorders, resulting in the abnormal accumulation of toxic free copper ions in the body, primarily deposited in the liver, central nervous system, kidneys, and cornea, causing a range of progressively worsening pathological changes, including liver cirrhosis, neuropsychiatric disorders, kidney damage, and the characteristic Kayser-Fleischer (K-F) ring in the cornea. Globally, the prevalence of WD is approximately 1 in 30,000 to 100,000 people, with a carrier rate of the pathogenic gene of about 1 in 90. The disease can affect individuals across a wide age range but is primarily concentrated in children and adolescents, with the highest incidence between the ages of 5 and 35. It is noteworthy that, according to epidemiological studies, the incidence and carrier rates of WD in Asian regions, including China, may be higher than in Western countries, with some data suggesting that the incidence in China may be close to one in ten thousand. In terms of treatment, hepatolenticular degeneration faces significant clinical challenges and unmet needs. The current standard treatment requires patients to undergo frequent drug interventions for a long time or even lifelong after diagnosis. However, these treatment methods 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 and late stages, posing a severe threat to patients' lives. Therefore, exploring more efficient treatment strategies that can fundamentally improve copper metabolism abnormalities is particularly urgent.
GC310 Adeno-Associated Virus Injection is an AAV gene therapy drug independently developed by Genecradle Therapeutics for the treatment of hepatolenticular degeneration. Non-clinical studies have shown good drug safety and significant efficacy. After a single treatment, the target tissue can express the biologically functional miniATP7B copper ion transporter protein, restoring copper ion metabolism and increasing ceruloplasmin levels, with the potential to significantly improve the status of the hepatolenticular degeneration community in the long term.