Making gene therapy affordable with droplet digital PCR

Gene therapy has the potential to bring relief to millions of individuals with diseases ranging from β-thalassemia to sickle cell disease across Asia and beyond.

Marwan Alsarraj, Biopharma Segment Manager, Digital Biology Group, Bio-Rad, US

This novel treatment modality is showing tremendous economic potential. According to one analysis, the global gene therapy market is expected to reach $3.3 billion by 2027 and enjoy a post-COVID-19 compound annual growth rate of 19.5 per cent.

However, gene therapy poses several unique technical challenges that must be overcome before becoming widely available and affordable. For example, gene therapy depends on inserting a healthy genetic sequence into a patient using a vector such as an adeno-associated virus (AAV), but it is difficult to produce a therapeutic batch that contains enough vectors for the therapy to benefit patients.

Upstream bioprocessing of AAV vectors often yields crude harvests containing too low vector genomes per milliliter, to create a potent therapy in a reasonable volume. Therapies that target smaller compartments in the body, such as the central nervous system, where the volume must be reduced, require a vector concentration that is much higher. This means developers must concentrate their AAV vectors 100-10,000 times.