Nobelpharma Receives Excellent Abstract Award from the Korean Pediatric Society

The research team at Nobelpharma, a bio-venture specializing in rare medicines, has been recognized for their academic excellence in the study of the rare disease mucopolysaccharidosis.

On the 20th, Nobelpharma announced that Jindonggyu, Nobelpharma’s Chief Scientific Officer (CSO) and Emeritus Professor at Sungkyunkwan University School of Medicine, along with Goara, Head of Nobelpharma’s Translational Research Division, received the Excellent Abstract Award at the 73rd Autumn Scientific Conference of the Korean Pediatric Society.

Professor Jindonggyu and Dr. Goara are jointly developing a new drug for Sanfilippo Syndrome Type A (MPS IIIA), one type of the rare disease mucopolysaccharidosis (MPS), in collaboration with GC Green Cross. The drug has been designated as an Orphan Drug (ODD) and Rare Pediatric Disease Drug (RPDD) by the U.S. FDA.

The Autumn Scientific Conference of the Korean Pediatric Society is an annual event where societies and researchers related to pediatric and adolescent diseases gather to present research status and papers, and to award academic, research, and paper prizes. This year, the conference was held over two days from October 19 at the Grand Walkerhill Seoul in Gwangjin-gu, Seoul, focusing on changes in pediatric research and treatment and the establishment of emergency medical systems.

At the conference, Professor Jin and Dr. Go, together with co-first author Aram Yang, Professor of Pediatrics at Kangbuk Samsung Hospital, submitted a research abstract titled "Therapeutic Potential of Intracerebroventricular Human Recombinant N-sulfoglucosamine Sulfohydrolase Enzyme Replacement Therapy in a Mucopolysaccharidosis IIIA Mouse Model." Through this, they disclosed the nonclinical efficacy of intracerebroventricular (ICV) enzyme administration (ERT) in Sanfilippo Syndrome Type A.

Mucopolysaccharidosis is a disease in which mucopolysaccharides (glycosaminoglycans) accumulate in the lysosomes of cells, causing symptoms in various organs throughout the body. Sanfilippo Syndrome Type A is a type of mucopolysaccharidosis caused by a deficiency of heparan-N-sulfatase. It manifests with severe intellectual disability, hearing and vision impairments, and other physical abnormalities, and in severe cases, leads to death around the age of 15. Currently, no new drugs have been developed for this condition.

A Nobelpharma official stated, "Since there is currently no appropriate treatment for Sanfilippo Syndrome Type A, most patients die at an early age due to worsening symptoms. We will accelerate research and drug development for Sanfilippo Syndrome Type A to lead efforts in alleviating the difficulties faced by patients suffering from this rare disease."

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