PHOENIX NEST, INC.

Identification of Potential Biomarkers and Clinical Tools for use in Mucopolysaccharidosis IIIC Patients.

Operating in national institute of neurological disorders and stroke based in BROOKLYN, NY, PHOENIX NEST, INC. was founded in 2012.

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PROJECT SUMMARY/ABSTRACT
Lysosomal storage diseases (LSD) are rare inherited metabolic disorders caused by defects in the cellular
catabolic system. Mucopolysaccharidosis Type IIIC (MPS IIIC or Sanfilippo disease type C) is one such LSD
that is caused by deficiency of the enzyme heparan sulfate acetyl CoA: -glucosaminide N-acetyltransferase,
(HGSNAT) essential for degradation of heparan sulfate, a repeating carbohydrate generally found attached to
proteoglycans. This disease causes accumulation of heparan sulfate resulting in a progressive and severe
neurological deterioration early in life with little somatic features. The symptoms in patients with MPS IIIC may
present at an average age of 3.5 years of age with psychomotor developmental delays and behavioral problems.
Before the age of 15 years verbal communication is often lost in patients with MPS IIIC. Most lose the ability to
walk between the 20 and 30 years of age. The condition is fatal by an average age of 34 years (range, 25-48).
Enzyme replacement therapies are not an option since the protein is localized and bound to lysosomal
membrane. There are currently no treatments available for treatment of MPS IIIC. Individuals affected by
MPS IIIC are managed with supportive care, consultation with medical professionals from multiple disciplines,
physical therapy, and pharmacological interventions to alleviate symptoms. Gene

Its activity falls under national institute of neurological disorders and stroke; the profile below is assembled from public directory data.

The company is recorded as being based in BROOKLYN, NY.

The company is listed at the SBIR/STTR stage of development.

Recorded headcount: 11-50 employees.

It has disclosed roughly $1.1M in funding.

Website: phoenixnestbiotech.com