Nobelpharma Recruits Rare Disease Experts from the US and Japan as SAB Members

[Asia Economy Reporter Hyungsoo Park] Nobelpharma, a bio-venture specializing in rare medicines, announced on the 22nd that it has recruited world-renowned scholars Professor Paul Harmatz of the University of California, San Francisco (UCSF) in the United States and Professor Torayuki Okuyama of Saitama Medical University as members of its Scientific Advisory Board (SAB).

It is expected that medical advisory with extensive experience and expertise in the field of rare diseases will be added to the research and development of treatments for rare diseases. They plan to receive advice on the design and implementation of global clinical trials starting next year.

Professor Paul Harmatz is an expert in mucopolysaccharidosis (MPS) who served for 12 years at Harvard Medical School in the United States. He has published over 200 papers on MPS type 2, MPS type 6, and other rare metabolic diseases. He has led clinical trials for the development of treatments for MPS and other lysosomal storage diseases (LSDs) and obtained approval from the U.S. Food and Drug Administration (FDA) for five new drugs. He led the clinical trials for Vimizim, a treatment for MPS type 4A, which is similar to MPS type 3A and MPS type 4B, types that Nobelpharma is currently researching.

Professor Torayuki Okuyama is currently a specially appointed professor at Saitama Medical University. He is an authority in the field of rare diseases, having served as chairman of the Japanese Society for Inborn Errors of Metabolism and as a clinical professor at Tokyo Medical and Dental University. He led clinical trials for intracerebroventricular (ICV) administration of Hunter syndrome treatment, which received approval from the Japanese Ministry of Health, Labour and Welfare.

Park Chan-ho, CEO of Nobelpharma, said, "With the addition of distinguished experts in pediatric rare diseases to the Nobelpharma SAB, we expect the pace of new drug development to accelerate."

The two scholars stated, "We will work closely with the Nobelpharma research team to develop and provide innovative treatments for rare diseases to patients who have no alternatives."

Nobelpharma is a bio-venture primarily developing treatments for rare pediatric diseases. It was founded by researchers who developed Hunterase, a treatment for rare diseases. Currently, it is developing innovative new drugs targeting ultra-rare diseases such as Sanfilippo syndrome type A, Morquio syndrome type B, GM1 gangliosidosis, and Krabbe disease.

This content was produced with the assistance of AI translation services.