Accelerating AAV Analytical Development And Testing With Advanced Methods And Comprehensive Solutions

By David Colter, PhD, and Dan Stringer, PhD, Center for Breakthrough Medicines

Advances made in the gene therapy space over the last several years have underscored the importance of accelerated innovation for its manufacturing. Adeno-associated virus (AAV) gene therapies represent a promising modality for the space, owing to a number of preclinical and clinical successes in the last few years related to AAV-mediated gene replacement, silencing, and editing. This work has resulted in the commercialization of four AAV gene therapies in the U.S. and EU – the spinal muscular atrophy treatment Zolgensma; Luxturna, which targets inherited retinal diseases; Upstaza, used to treat children with aromatic l-amino acid decarboxylase (AADC) deficiency; and the hemophilia drug Roctavian.

These drugs represent life-changing intervention for patients. As more AAV gene therapies progress through the pipeline into clinical trials, enabling their successful development and commercialization will be critical to bringing more cures to patients. Testing drug product is a core component of that enablement and while many strides have been made in bringing additional capacity online for the manufacturing of AAV gene therapies, a persistent lack of comprehensive, optimized testing capabilities has created a landscape where testing backlogs, lengthy method transfer timelines, and large sample volumes needed are the norm. Finding a contract development and manufacturing organization (CDMO) that can perform all the necessary testing for an AAV product, while preserving precious drug product and adhering to or accelerating timelines, can make a significant difference for a nascent company or novel drug.