On November 14, 2024, Genecradle Therapeutics' independently developed new product, GC310 adeno-associated virus (AAV) injection, was accepted for review by the National Medical Products Administration for clinical trial registration. Classified as a Class 1 therapeutic biological product, it is indicated for the treatment of hepatolenticular degeneration. This marks the fourth gene therapy drug product that Genecradle Therapeutics has submitted for a new drug clinical trial following GC101, GC301, and GC304.

▲ Image Source: Screenshot from the official CDE website

GC310 Adeno-Associated Virus Injection

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.

Hepatolenticular Degeneration (Wilson Disease, WD)

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, and the carrier rate of the pathogenic gene is 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 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 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.