Gene Editing Breakthroughs: A New Hope For Patients

By Anshul Mangal, President of Project Farma and Precision ADVANCE, and Cynthia Pussinen, Advisor & Executive Consulting Partner

Gene therapies have rapidly evolved over the past decade, and the rapid genome sequencing and editing offered by CRISPR Cas-based gene editing treatments are spurring advancements in disease research and treatment. CRISPR is now capable of altering multiple DNA sequences or base pairs at once in a technique called multiplex base editing.

Base editing is advantageous compared to other historical methods of editing, due to its ability to be precise and specific while leaving the DNA intact, thereby eliminating the need for complex repair mechanisms. The ability to alter a single base pair also provides an opportunity to solve for genetic diseases without targeting a genetic mutation sequence and can address the core problem or protein.

These recent breakthroughs in gene editing technology hold the potential to dramatically improve outcomes for patients. However, researchers still face a number of obstacles and are working aggressively to identify roadblocks and find solutions in therapeutic development, delivery, and accessibility. Explore how these innovative technologies hold the potential to create meaningful change for patients living with genetic diseases and the obstacles developers are navigating on the path to commercialization.