By Florence Vicaire, GE, Joe Makowiecki, GE, and Carol Houts, Germfree
With the exciting promise of addressing previously unmet medical needs, the rapidly growing field of gene therapy has already begun to revolutionize patient treatment. The ability to create a genetic fix for certain cancers and other rare diseases has resulted in the launch of several innovative gene therapy drugs over the last few years, such as Kymriah™, Luxturna™, and Zolgensma™. Significant growth is expected to continue, with the FDA anticipating the approval of 10 to 20 cell and gene therapy products a year by 2025. (1) This belief is supported not just by the clinical success rate of these drugs but also the current pipeline, which includes close to over 700 clinical trials within the gene therapy field, nearly doubling the amount only a few years ago in 2016 (400) (based on an internal analysis by GE Healthcare Life Sciences, 2010 to 2019).
A key component of gene therapy is the use of viral vectors for in vivo and ex vivo gene transfer, which are used in approximately 70 percent of cell and gene therapies under trial. (2) Producing these gene delivery vehicles, whether they are used to carry a therapeutic gene to a target cell or to initiate cell transduction, relies on specialized skillsets and effective scale-up manufacturing strategies.
Uncertainties around the long-term durability of gene therapy development and manufacturing historically permeating the industry, though, have led to a field of drug companies and contract development and manufacturing organizations (CDMOs) that are unprepared for the market boom of regenerative therapies, resulting in a shortage of viral vectors to meet today’s demand.