Article | December 26, 2023

High-Velocity Development: Gene Therapy Vs. Small Molecule

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

GettyImages-1386852216 cell therapy

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.

access the Article!

Get unlimited access to:

Trend and Thought Leadership Articles
Case Studies & White Papers
Extensive Product Database
Members-Only Premium Content
Welcome Back! Please Log In to Continue. X

Enter your credentials below to log in. Not yet a member of Advancing RNA? Subscribe today.

Subscribe to Advancing RNA X

Please enter your email address and create a password to access the full content, Or log in to your account to continue.

or

Subscribe to Advancing RNA