Small Biotech Gets $1.7 Million to Develop Treatments for Sanfilippo (MPS III)

Ruth J Hickman, MD

Phoenix Nest, Inc will receive over $1.7 million in grant funding recently authorized by the National Institute of Neurological Disorders and Stroke (NINDS). The biotech company focuses solely on research into treatments for different forms of the rare genetic disorder MPS III, also known as Sanfilippo disease. With the grant funds, the company will continue its partnership with the Los Angeles Biomedical Institute (LA BioMed).

About Sanfilippo Disease

Sanfilippo disease (MPS III) is a member of a larger group of disorders called mucopolysaccharidoses (MPS). In these disorders, the body cannot break down certain sugar molecules. In MPS III, the body cannot break down a specific long chained sugar molecule, heparan sulfate. Due to genetic defects, the enzymes needed to breakdown the molecule are defective or absent. MPS III is further broken down into four subtypes, A-D, based on the specific enzyme that is affected.
In each type of MPS III, Heparan sulfate builds up, damaging cells in multiple parts of the body. This leads to the symptoms of the progressively worsening condition. These include mental disability, behavioral problems, coarse facial features, muscle weakness, and sleep difficulties. There is currently no treatment available. Eventually most patients die in their teens or early twenties.

Phoenix Next

Jill Wood co-founded Phoenix Nest in 2012 to provide the infrastructure for treatment developments for MPS III, which affects her son. She began the company to ensure that potential treatments for MPS III would receive would receive prompt investment, even if no other pharmaceutical company was interested. In an interview with Rare Disease Report the patient advocate turned biotech executive explained, “Phoenix Nest is about the families. It’s not about big money. It’s about providing treatment for our children.”
Phoenix Nest plans to put the research grant to good use, as it develops potential breakthrough therapies. Patricia Dickson, MD, is LA BioMed lead researcher and the director of the institute’s MPS Research Laboratory. She said in a press release, “After promising results obtained in our first preclinical studies for an enzyme replacement for MPS IIID, we will next look at efficacy in a disease model with a goal of moving towards the clinic as quickly as we can.”
Sean Elkins, PhD, the chief executive officer of Phoenix Nest, Inc., explained, “The research is seeking to a develop a therapy that will limit or reverse the neurological damage caused by MPS IIID by delivering an enzyme, recombinant human alpha-N-acetylglucosamine-6-sulfatase (rhGNS), intrathecally to effectively treat the underlying causes of the neurological symptoms of MPS IIID.”
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