Phoenix Nest Secures $2.9M Grant for Sanfilippo Syndrome Gene Therapy

The AAV9 gene therapy, currently in development, aims to address the underlying genetic causes of Mucopolysaccharidosis type III. CEO Jill Wood, who founded the company after her own child was diagnosed with the condition, credited the National Institutes of Health and the National Institute of Neurological Disorders and Stroke for their sustained support of the program. The company is actively enrolling patients into a natural history study at the University of Texas Southwestern in Dallas to build upon their clinical data.

The FDA has already granted the therapy orphan drug and rare pediatric disease designations, accelerating its path toward potential commercialization. Beyond manufacturing efforts, the company is utilizing a specialized application called C-RARE to track daily patient functionality. Researchers are also leveraging Heparan Sulfate as a surrogate biomarker to measure the treatment's efficacy, aiming to move the therapy from the lab to clinical application for children facing this rare disorder.