Phoenix Nest, a Brooklyn, New York-based biotechnology company, has received a USD 1.49 million NIH SBIR grant to fund manufacturing and analytical development of JLK-247, a gene therapy candidate for Mucopolysaccharidosis Type IIIC (MPS IIIC, also known as Sanfilippo syndrome type C). The award, issued by the National Institute of Neurological Disorders and Stroke, will support production of a 500-liter current good manufacturing practice (cGMP) batch of vector ahead of a planned Phase I trial.
MPS IIIC is caused by deficiency of the enzyme HGSNAT, leading to heparan sulfate accumulation and progressive neurodegeneration in children, with death typically occurring by the mid-30s. Because the deficient enzyme is membrane-bound within lysosomes, it is not amenable to conventional enzyme replacement therapy, leaving gene therapy as one of few viable modalities. Phoenix Nest has reported a positive prior interaction with the FDA regarding its clinical development path. Most of the funded work—vector scale-up, analytical assay qualification, and stability and sterility testing—will be executed through a contract development and manufacturing organization rather than in-house.
There are currently no approved therapies for MPS IIIC, and the company describes its program as a candidate for the first indication-specific treatment, though no clinical efficacy or safety data have yet been generated. The grant reflects continued NINDS support for late-preclinical manufacturing readiness in rare pediatric neurodegenerative disease, a funding category that has grown as more small biotechs advance viral vector programs toward IND filing. For Phoenix Nest, led by principal investigator Srikanth Singamsetty, the award marks a concrete step toward initiating first-in-human dosing for an ultra-rare disease with no existing disease-modifying options.
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