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.