- Andelyn Biosciences will manufacture clinical-grade adeno-associated virus (AAV) for Hubble Therapeutics’ treatment of Leber Congenital Amaurosis 16 (LCA16).
- The AAV production will support Phase 1/2 clinical trials aimed at addressing this rare genetic eye disease.
Andelyn Biosciences, Inc., a contract development and manufacturing organisation (CDMO) specialising in cell and gene therapies, has been selected by Hubble Therapeutics to manufacture adeno-associated virus (AAV) for the treatment of Leber Congenital Amaurosis 16 (LCA16), a rare and severe genetic retinal disorder. The manufacturing will be done using Andelyn’s suspension AAV Curator™ Platform, which will generate GMP-grade material for upcoming clinical trials.
LCA16 is an early-onset retinal disease caused by mutations in the KCNJ13 gene, leading to severe visual impairment and eventual blindness. Hubble Therapeutics’ treatment, HUB-101, aims to introduce normal copies of the KCNJ13 gene using AAV technology. The programme has received both Rare Pediatric Disease and Orphan Disease Designations from the FDA. Andelyn will optimise and scale up the production process, including generating GMP-grade plasmids, for use in Phase 1/2 clinical trials.
Jeff Sabados, President of Hubble Therapeutics, commented on the partnership: “Over the past 18 months, we have identified best-in-class partners like Andelyn Biosciences to help us advance our gene therapy programme for LCA16. We are grateful for Andelyn’s commitment to supporting our efforts to treat this devastating genetic condition.”
Matt Niloff, Chief Commercial Officer of Andelyn Biosciences, added: “We are honoured to play a role in the development of HUB-101 and contribute to the potential clinical benefits of this therapy. Our work in the CDMO space continues to support innovative gene therapies aimed at transforming patients’ lives.”
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