Danaher Corporation

02/08/2024 | Press release | Distributed by Public on 02/08/2024 07:05

IDT’s Rolf Turk considers the future of CRISPR

There's a long-running saying in the gene therapy field that also applies to CRISPR in general, and it's that the three biggest challenges are "delivery, delivery, and delivery." Identifying the "ZIP code" for targeting cells and getting the therapies to the right tissues in the body, plus having the ability to deliver larger payloads, could open a lot of doors eventually.

I think we also really need to figure out the genetic variability between different populations and how that may impact efficacy and safety. With the lower and lower cost of sequencing, we have the opportunity to understand biological variation from one person to another, and how it changes during development and with age.

We also need to be thinking about scaling up. If we want to target indications with much larger patient populations, we will need a more robust and standardized manufacturing process. And of course we have to think about cost and access - now is the time to set the stage for more accessible treatments that can benefit large numbers of people in a wide range of settings.

Essentially, we need standardization that would allow for quick approvals, an increased likelihood of cures, and a reduction in cost. But we also need to remember that every cell type is different and might require some tweaks to get the right CRISPR "recipe." If we can achieve both of those goals, CRISPR-based therapies might well someday become a first choice for treating patients.