Realising the potential of AAV gene therapies

Through pre-clinical and clinical trial success, adeno-associated viral vectors (AAVs) have consistently demonstrated their effectiveness as a tool for gene delivery to treat a variety of human diseases.

As we stand at the precipice of an era that truly embraces gene therapies, there are clearly still a number of challenges to overcome to fully realise the potential of innovative AAVs. Addressing the burden of cost through improving scalability will be instrumental in broadening patient access to new cell and gene therapies (CGTs). Achieving this in the relatively new and dynamic CGT regulatory landscape will rely on flexible and agile developers and manufacturers.

AAVs evolving the in vivo gene therapy space

In 2012, the European Medicines Agency (EMA) approved the first AAV gene therapy, Glybera (Alipogene tiparvovec). This revolutionary in vivo therapeutic was designed to treat a rare recessive disorder – lipoprotein lipase deficiency (LPLD) – which can cause severe pancreatitis. Since Glybera’s approval, two more AAVs have come on the market targeting neurological and ophthalmological diseases. The success of these therapies in providing once unimaginable patient outcomes has spurred the biopharma industry to take on the challenge of developing additional AAV products. In September 2022, the Alliance for Regenerative Medicine (ARM) reported that for all ongoing gene therapy clinical and CBIO trials, AAV ranks second among vectors used, and was the primary vector for gene therapy trials.¹