JLK-247rp

JLK-247rp is an experimental adeno-associated virus serotype 9 (AAV9) vector-mediated gene therapy being developed by Phoenix Nest for the treatment of retinitis pigmentosa associated with mutations in the heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT) gene. The therapy is an ocular-specific expansion of JLK-247, which is designed for intrathecal administration to treat Sanfilippo syndrome type C (Mucopolysaccharidosis Type IIIC). JLK-247rp is administered via intraocular injection (such as intravitreal or subretinal delivery) to deliver a functional copy of the HGSNAT gene directly to the retina, restoring HGSNAT enzyme activity and preventing the accumulation of heparan sulfate that leads to retinal degeneration.