Much Needed Funding Supports Gene Therapy Manufacturing for Children with Sanfilippo Syndrome Type C
BROOKLYN, N.Y., Aug. 5, 2026 /PRNewswire/ — Phoenix Nest is a thrilled recipient of the Small Business Innovation Research (SBIR) grant [R44NS147802; $2,984,658] that will fund manufacturing of clinical grade product to treat Sanfilippo syndrome type C or Mucopolysaccharidosis type III (MPS III) patients. This AAV9 gene therapy, JLK-247, would be the first treatment and a potential cure for this rare and fatal childhood disease.
Pheonix Nest CEO and parent of child living with MPS IIIC, Jill Wood said: “I want to thank the NINDS leadership and NIH reviewers for believing in our program over the years with this grant and previous grants for nonclinical and clinical development. We are currently recruiting MPS IIIC patients into our natural history study [NCT05825131] at University of Texas at Southwestern (UTSW, Dallas, TX). This newly awarded grant supporting our manufacturing moves us another step closer to our goal of delivering therapy to the patients.”
Phoenix Nest has obtained an orphan drug designation and rare pediatric disease designation from the FDA for the JLK-247 gene therapy. Phoenix Nest has developed a video recording application (C-RARE) to capture daily living and function in the MPS IIIC patient population. The recent developments in establishing Heparan Sulfate as a surrogate biomarker will aid in commercializing this life altering treatment.
About Phoenix Nest
Phoenix Nest is a unique biotech company founded by patient caregivers and scientists, dedicated to developing treatments for children with Sanfilippo syndrome. Funding has come from patient organizations and NIH SBIR grants [5R44NS137892, 5R44NS129393].
Inspired by the mythical Phoenix, which built its nest with healing herbs, Phoenix Nest reflects parents’ efforts to fill their homes with medicinal treatments, aiming for their children to eventually outgrow the “nest.”
Phoenix Nest would like to acknowledge support from patient organizations: Columbus Children’s Foundation, Cure Sanfilippo Foundation, International Sanfilippo Syndrome Alliance, Upstream Genes, and Vanicre Les Maladies Lysosomales.
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SOURCE Phoenix Nest Inc.
