Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Q1 2023 Earnings Call Transcript

Those same cells, the VX-880 cells are the foundation for Program 2, which is the self-plus device program, and those same cells are the foundation for Program 3, which is the hypoimmune program. And that’s really important because — as you know, there are two parts to this. The first is having cells that are fully differentiated and insulin producing. We’ve got that. And the second is how to evade the immune system. In the 264 program, we made the immune system with a device. I’m not going to share any more details than we have in the past. But I will say that it’s a proprietary device that has a particular material, geometry, and structure that allow for oxygenation and nutrient transport as well as sensing of glucose and release of insulin without worry of the immune system coming into the cells and from what we’ve seen to date in large animal models and small, no fibrosis.

And of course, the third is the cells with the edits so that the edited cells evade the immune system. With regard to when we can see data from VX-264, we haven’t guided to that, but we are very pleased with the clearance of the CTA and IND and we expect the trial to start dosing patients in the near future. With regard to the DMD program, you’ll recall that our approach is different than the approach being taken by many in the field. That is to say our approach is grounded in human genetics and what we see is that if you have near full length or full-length dystrophin, which is the goal of our program, you have a very mild version of the disease. There is no such human genetic validation for an approach using microdystrophin. We’re excited about the program.

It is in its IND-enabling studies, and we are on track to complete those studies and get the IND filed towards the second half of this year.

Susie Lisa: Thanks, Colin. Just that brings us to time. So can you wrap it up, please?

Stuart Arbuckle: Yes, ma’am. That will conclude our question-and-answer session as well as our conference call for today. A replay of today’s event will be available shortly after the call concludes by dialing 1 (877) 344-7529 or 1 (412) 317-0088 and you can use the replay access code 8384718. Everyone, have a great day.