Gene Editing Your Cell Therapy: Key Considerations for Biopharma Companies and Investors

Cell therapy has become an important pharmaceutical asset class in recent years, in part due to the discovery of T-cell receptors (“TCRs”) and the development of chimeric antigen receptors (“CARs”). The clinical promise of CAR-T cell therapy has attracted both scientific and financial interest as companies, universities, researchers and clinicians work to harness the immune system and engineer novel CAR-T cell therapies to address a number of high unmet medical needs. Today there are more than 130 commercial and more than 30 academic organizations developing CAR-T cell therapies across all phases of development, accounting for approximately 13 percent of all players working in the cell and gene therapy space.

Despite this surge in interest, many cell therapy products come with significant safety and toxicity issues and the crowded market makes differentiation an increasing challenge. As a technology, gene editing opens a number of possibilities in the development of novel, safe and effective immuno-oncology cellular therapies. Shown to be effective in generating safer autologous CAR-T products, gene editing can be a key component of any cell therapy strategy.

Companies considering the potential of gene editing to improve or augment their portfolios have at their disposal a range of gene editing tools, including ZFNs, TALENs, CRISPR/Cas9, “base editing”, and CRISPR-Cas13 (for RNA editing). Before committing to any gene editing in any product development strategy, however, it is important to ask critical questions, such as: