COLUMBUS, Ohio , Oct. 9, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc. , a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by Hubble Therapeutics, LLC, to manufacture adeno-associated virus (AAV) using its suspension AAV Curator TM Platform to manufacture clinical grade AAV for the treatment of Leber Congenital Amaurosis 16 ("LCA16").
LCA16 is a severe, early-onset retinal dystrophy resulting from mutations in the KCNJ13 gene, which expresses a critical ion channel in the retinal pigment epithelium cells in the retina. Children typically present with visual abnormalities within five years after birth, followed by progressive retinal degeneration during the next two decades of life, ultimately resulting in blindness. HUB-101 has received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA for this program.
Andelyn will optimize and scale-up the HUB-101 process, generate GMP-grade plasmids, and produce GMP vectors that include normal copies of the KCNJ13 gene. From this partnership, Hubble Therapeutics LLC ("HubbleTx") will be able to conduct Phase 1/2 clinical trials with the intention to demonstrate safety and efficacy in an adult patient population. "Over the past 18 months we have identified best-in-class partners including Andelyn Biosciences to establish a gene therapy development program for adult and pediatric patients suffering from Leber Congenital Amaurosis 1.