7:23 PM
May 17, 2019
 |  BioCentury  |  Product Development

AAVs on the brink: Charting the clinical landscape of in vivo gene therapies

BioCentury maps the world of clinical AAV gene therapies to the companies testing them and their in vivo indications

Gene therapy is finally teetering on the edge of becoming a drug development mainstay, with at least nine AAV-based therapies in Phase III or II/III testing. An analysis of the AAV therapies in the clinic reveals a growing diversity of vector subtypes being used in vivo, engineering strategies gaining traction, and an expansion of the modality beyond monogenic diseases.

The 2017 approval of Luxturna voretigene neparvovec-rzyl from Spark Therapeutics Inc. as the first in vivo gene therapy broke the modality’s twenty-year logjam.

Now, over 70 gene therapies are in proof-of-concept or pivotal trials, which will answer whether the modality can deliver on its promise.

Like Luxturna, many of them deliver therapeutic genes via adeno-associated viral (AAV) vectors, due to the vectors’ ability to infect non-dividing cells and their comparative safety. AAVs integrate into a specific site in the genome, avoiding the random integration of retroviruses -- and lentiviruses that can cause cancer. They also have relatively low immunogenicity, an advantage over adenoviruses.

BioCentury plotted the rise and diversification of AAVs in the clinic, mapping the different vectors to the companies that are testing them and the indications they aim to treat.

On the verge of answers

Between 2009-2018, the number of new clinical trials started each year of in vivo gene therapies rose more than sixfold, according to BioCentury’s analysis of data from ClinicalTrials.gov.

Activity hovered around five new trials annually until 2014, when a ramp-up began, with a particularly big jump between 2016 and 2017, possibly a response to the approval of GlaxoSmithKline plc’s Strimvelis, an ex vivo gene therapy to treat ADA deficiency. The therapy, which uses a retroviral...

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