High-Velocity Development: Gene Therapy Vs. Small Molecule

By Raul P. Lima, Executive Vice President, Clinical Operations, inSeption Group

While gene therapy has been around for decades, the field is not as mature as many might imagine. Additionally, the pace, structure, and funding dynamics for gene therapy development are unique among treatment modalities. Combined with the inherent difficulty of the chemistry behind creating a gene therapy, these factors undermine countless development efforts and, by extension, sink many biotechs.

In gene therapy, clinical tasks overlap and translational medicine digs deeper and takes longer. Rather than sequential development, many activities take place in parallel. That crunch on chemistry, manufacturing, and controls (CMC) personnel, as well as lab personnel, is difficult to navigate. The sponsor does not know how to make the drug and does not have full release on it. Nor does the sponsor have all the data it would like to have heading into the first-in-human patient study (Phase 1/2). Thus, a carefully plotted approach is required to minimize risk and maximize chances for success.