GC Biopharma and Novel Pharma Launch Clinical Trial for Sanfilippo Syndrome Treatment

Launch of Phase I Trial for MPS IIIA Treatment

GC Biopharma and Novel Pharma have taken a significant step in the quest for effective treatments for genetic disorders with the commencement of a global Phase I clinical trial for a new drug, 'GC1130A'. This innovative therapy targets Sanfilippo syndrome type A (MPS IIIA), a severe genetic disorder that primarily affects the central nervous system.

On November 25, 2024, it was announced that the first patient in the United States has received the dosing in this multinational clinical trial, a crucial milestone for both companies involved. MPS IIIA is characterized by the accumulation of heparan sulfate in the body, leading to debilitating neurological issues and typically resulting in death around the age of 15. Currently, there are no approved treatments available, highlighting a significant unmet medical need that this trial aims to address.

The clinical trial has garnered Investigational New Drug (IND) clearance in the US, Korea, and Japan, paving the way for its execution at several esteemed institutions. In the United States, the University of California, San Francisco (UCSF) Benioff Children's Hospitals will participate alongside hospitals in Korea and a site in Japan.

Participants, aiming to recruit children aged between two and six diagnosed with MPS IIIA, will undergo surgery to implant a device that allows administration of 'GC1130A' directly into the cerebrospinal fluid via intraventricular injection. The regimen involves dosing every two weeks over the course of two years, during which the researchers will assess the treatment's safety, tolerability, and efficacy.

The innovative approach of 'GC1130A' utilizes GC Biopharma's proprietary technology to produce high-quality recombinant proteins that bypass the blood-brain barrier— a complicating factor in treating central nervous system disorders. In fact, previous non-clinical studies have demonstrated that the intracerebroventricular (ICV) delivery method of the treatment can be significantly more effective than traditional injection methods.

SooKyung Shin, who leads the Medical Division at GC Biopharma, underscored the importance of this initiative, stating that the dosing of the first patient represents a significant advancement in the global clinical trial process for 'GC1130A'. The company is committed to expediting the clinical development of this important treatment option.

The launch of this trial marks a hopeful beginning for those affected by Sanfilippo syndrome type A. With the expertise of both GC Biopharma, a major player in the biopharmaceutical domain, and Novel Pharma, a biotech company dedicated to rare pediatric diseases, the research community is looking towards potential breakthroughs in treatment modalities that can significantly improve living conditions for young patients afflicted by this devastating disorder.

As the trial progresses, the focus will remain not only on the drug's effectiveness but also on the broader impacts it may have on the quality of life for children diagnosed with MPS IIIA, and on the future of treatment strategies for rare genetic diseases.