Phoenix Nest Inc., Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and Washington University in St. Louis Announce a New NIH Grant for Sanfilippo Syndrome (MPSIIID)

The National Institute of Neurological Disorders and Stroke at the National Institutes of Health (NIH) recently awarded $5,612,276 in SBIR Phase IIB grant funding to Phoenix Nest, Inc., The Division of Medical Genetics at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and Washington University in St. Louis to develop a therapy for Sanfilippo syndrome (also known as Mucopolysaccharidosis IIID, (MPSIIID)). This is a devastating neurodegenerative lysosomal storage disorder of childhood for which there is no cure or effective treatment available.

Dr. Patricia Dickson (Division Chief, Genetics and Genomic Medicine, Washington University in St. Louis) states, “Dr. Tsui-Fen Chou (Assistant Professor of Pediatrics, LA BioMed), Phoenix Nest Inc, and myself have collaborated over the last four years to develop an enzyme replacement treatment for MPS IIID that shows promise in an animal model of the disease. Our strategy proposes to deliver the recombinant human alpha-N-acetylglucosamine-6-sulfatase (rhGNS) as an enzyme replacement therapy to treat patients. This grant will allow us to develop significant quantities of protein and test its safety prior to a clinical trial.”

“Enzyme replacement therapy, giving patients a working enzyme to replace a mutated or missing one in the body, is a proven strategy that has been applied to several lysosomal storage diseases over the last 20 years. We are quite literally standing on the shoulders of these scientists who have led the way and demonstrated that it is possible,” said Dr. Tsui-Fen Chou.

“We are excited to continue our collaboration as we develop this enzyme replacement further with the support of the NIH. Our ultimate goal is to commercialize this treatment and ensure it reaches the patients we know are out there waiting for it,” stated Dr. Sean Ekins, CEO of Phoenix Nest Inc.

“Phoenix Nest Inc. was founded by Sanfilippo families to develop treatments for the different forms of Sanfilippo syndrome. These terrible illnesses cause enormous suffering for children and our families. As a parent of a child with this syndrome, I know this only too well. We are indebted to researchers like Dr. Dickson and Dr. Chou and their institutes for supporting this important work and progressing it so rapidly,” stated Ms. Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest.

About Phoenix Nest, Inc. - Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome founded by and solely owned by several Sanfilippo Syndrome families. They actively collaborate with academic researchers to fund and commercialize their technologies. Phoenix Nest, Inc. also licenses assets from companies and universities that may have utility for Sanfilippo Syndrome. For more information, please visit

For further information, please contact Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest, 347-689-2186

About LA BioMed

LA BioMed is an incubator of innovation with a global reach and a 66-year reputation of improving and saving lives. Driven by the positive social impacts of breakthrough therapies, the Southern California-based research institute, and incubator – with over 100 principal investigators (PhDs, MDs and MD/PhDs) working on over 600 research studies, including therapies for multiple orphan diseases – is an essential element to one of the nation’s top-ten biotech clusters. Find out more at

Contact information Washington University School of Medicine in St. Louis

Judy Martin Finch, director of media relations



Research reported in this publication was supported by the National Institute of Neurological Disorders and Stroke of the NIH under Award Number R44NS089061. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

Source: Phoenix Nest Biotech