How a collaborative project aims to cut the cost of gene therapy
Stephanie Sutton |
Cobra Biologics, Pall, and the UK’s Cell and Gene Therapy Catapult have won a shared grant of £1.5 million from the UK’s innovation agency, Innovate UK, to investigate the feasibility of continuous manufacture of adeno-associated viruses (AAV) for gene therapy applications. Tony Hitchcock, Technical Director of Cobra Biologics, tells us more.
Could you tell us about the focus on AAV?
AAV is currently the main vector used to treat monogenetic diseases. The approach has shown spectacular results in the treatment of a broad range of conditions ranging from ophthalmic conditions through to whole body diseases, such as hemophilia and spinal muscular atrophy (SMA). We have seen the licensing of Luxturna for the treatment of retinal disease caused by the RPE 65 gene and it is anticipated that a number of other products, currently in phase III studies, will be successfully licensed in the coming months.
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