Participating as a sponsor in the US ‘Advance 2024’ conference
GC Pharma jointly develops treatment with Nobel Pharma
Promoting development as a direct intracerebroventricular (ICV) drug
ICV excellent drug delivery effect confirmed through non-clinical studies
GC Green Cross, together with Nobel Pharma, disclosed the status of clinical progress of an innovative new drug being developed as a treatment for the rare disease Sanfilippo Syndrome (Type A).
GC Green Cross announced on the 31st that it participated as a partner sponsor in ‘ADVANCE 2024’, a Sanfilippo community conference hosted online by the Cure Snfilippo Foundation, the largest Sanfilippo Syndrome patient association in the United States. At the conference, GC Pharma announced non-clinical research results and the status of ongoing clinical trials for ‘GC1130A’, an innovative new drug for Sanfilippo Syndrome Type A (MPS ⅢA) being jointly developed with Nobel Pharma.
Advance 2024 is an international event held online. Patients, families, clinicians, pharmaceutical companies, etc. will participate to share the latest knowledge related to Sanfilippo disease, present topics, and discuss clinical trials. In the case of GC1130A, a phase 1 clinical trial (IND) was recently approved in the United States. Ahead of the full-scale clinical trial, it received a lot of attention from clinicians, patients, and their families.
Sanfilippo syndrome type A is a recessive genetic disease in which heparan sulfate accumulates in the body due to a genetic defect, causing gradual damage. It is a rare, severe disease whose main symptom is severe brain damage and most patients die around the age of 15. Because there is no approved treatment yet, there is a huge unmet medical need.
GC1130A is being jointly developed with Nobel Pharma with the concept of applying GC Green Cross’ highly concentrated protein preparation technology, which can be administered to the central nervous system, to a treatment for Sanfilippo Syndrome. It is attracting attention as a first-in-class bioinnovative new drug. Development is being done using the ICV (Intracerebroventricular injection) method, which increases treatment effectiveness by injecting the treatment directly into the ventricle. It is similar to ‘Hunterase’, GC Green Cross’ Hunter syndrome treatment. Hunterase, developed through ICV, has obtained product approval in Japan.
At this conference, GC Green Cross and Nobel Pharma announced through non-clinical tests that delivering drugs through direct intracerebroventricular (ICV) administration can be expected to be up to 47 times more effective than direct intrathecal administration (IT). announced that it had been confirmed. The global phase 1 clinical trial currently underway will focus on confirming safety, tolerability, and effectiveness.
Shin Soo-kyung, head of GC Green Cross Medical Division, said, “Based on our experience and knowledge in developing treatments for MPS disease, we will do our best to contribute to improving the quality of life for patients with Sanfilippo Syndrome Type A.”
Kim Min-beom, Donga.com reporter [email protected]
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GC Green Cross has partnered with Nobel Pharma to develop an innovative treatment for Sanfilippo Syndrome Type A, a rare genetic disease. In a recent presentation at the ‘ADVANCE 2024’ conference organized by the Cure Sanfilippo Foundation, GC Green Cross shared updates on their drug candidate, GC1130A. This treatment is notable for its delivery method, utilizing intracerebroventricular (ICV) injection, which has been shown to be significantly more effective than intrathecal (IT) administration in non-clinical studies.
Sanfilippo syndrome type A is characterized by the accumulation of heparan sulfate, leading to severe neurological damage, with most patients not surviving past their teenage years. Currently, there are no approved treatments available, making GC1130A a potentially groundbreaking option for patients suffering from this condition.
The development of GC1130A, which employs advanced protein preparation technology from GC Green Cross, follows the successful model of their previous drug, Hunterase, also delivered via ICV, which has been approved in Japan. The ongoing Phase 1 clinical trials in the U.S. aim to establish the drug’s safety and effectiveness, drawing significant interest from the medical community and affected families.
Shin Soo-kyung, head of the Medical Division at GC Green Cross, expressed commitment to enhancing the quality of life for patients with Sanfilippo Syndrome Type A, leveraging their expertise in treating mucopolysaccharidosis (MPS) diseases.