Andelyn Biosciences has been selected by Hubble Therapeutics to manufacture clinical-grade AAVs.
The adeno-associated viral product will be used to treat leber congenital amaurosis, (LCA16) which is a severe form of retinal dystrophy that eventually leads to blindness.
Through the agreement, CDMO Andelyn will be responsible for the optimisation and scaling of the HUB-101 candidate, which has already received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA.
Andelyn will also be responsible for the generation of GMP-grade plasmids and vectors that contain normal copies of the KCNJ13 gene — the one affected in this rare disease.
From this collaboration, Hubble Therapeutics will have access to a clinical trial supply, allowing them to enter Phase I/II trials in an adult patient population.
Andelyn currently has three facilities located in and near Columbus, Ohio which offer end-to-end development and manufacturing capabilities for the cell and gene therapy sector.
President of Hubble Therapeutics, Jeff Sabados, commented: “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 16 (LCA16),”
“Andelyn Biosciences has become a global leader in gene therapy development for ultra-rare and orphan diseases and we are grateful for their unwavering commitment toward helping Hubble Therapeutics realize our steadfast promise to support families throughout the world impacted by this devastating genetic disease.”
Andelyn Biosciences' Chief Commercial Officer, Matt Niloff, added: “We are extremely grateful for the opportunity to produce the clinical grade material for HUB-101 and be a critical part of the pathway for Hubble Therapeutics to demonstrate clinical benefit for the LCA16 programme."
"We are thrilled that our establishment as a clinical and commercial CDMO is helping to accelerate so many programmes like HUB-101, which offer real hope to patients stricken with debilitating diseases.”