CRISPR Therapeutics AG Morgan Stanley 24th Annual Global Healthcare Conference

Thank you for having us.

Yeah, happy to. 2026 has been a great year for us so far as we move to the next phase of the company. What we have are several layers of value. One is CASGEVY, which is our commercial product for sickle cell and thalassemia, which we are partnered with Vertex on, and that's showing a very nice trajectory from a revenue standpoint, and we expect that to be a multibillion-dollar opportunity. The second is three assets that could potentially be ready for phase III next year or pivotals next year, and these are namely CTX310, which targets ANGPTL3. This is for a one-shot reduction of cardiovascular risk by using CRISPR-Cas9 to reduce the ANGPTL3. We have zugo-cel, which is an allogeneic CAR T, which is off-the-shelf, very convenient, and it could do a one-time immune reset for various B cell-driven autoimmune diseases.

The third being CTX611, which is our siRNA, which we are partnered with a company called Sirius on for Factor XI, which has the promise of anticoagulation without the risk of bleeding. That can be applicable in many different settings and indications, such as stroke prevention. So those three assets represent the next layer of value for us. Then we have two early assets in the clinic now. One is for A1AT, which is our first advanced editor, which has leapfrogged Gen 2 editors to be something very efficient and potent and very precise. We call it SyNTase editing, and that is going to dose patients soon. CTX340, which is for refractory hypertension, where we actually target angiotensinogen, and that can result in a pretty meaningful reduction in blood pressure in patients very soon after being edited. Those two will have data in the not-too-distant future.

So we have other programs beyond that. One of them is CTX321 for LPA, which kind of hangs in balance based on Horizon data, and also Type 1 diabetes. The last layer of value for us is our very prolific and productive research arm. We have a best-in-class in vivo CAR T now, both in the form of a transient CAR T based on mRNA, but also permanent CAR Ts based on editing, where we insert the CAR into cells, and that is permanent. We have in vivo HSC editing, where we directly edit hematopoietic stem cells, which can be durable, and many other things that we are working on. So all in all, a very rich portfolio, but we have the strong balance sheet to move all these programs forward ourselves and really change the face of medicine along many different dimensions.

Yeah, it is a great tailwind for us on CASGEVY. Vertex, in their last disclosure, announced revenues of about $76 million for the quarter, and also, more importantly, said that they are initiating over 100 patients per quarter. That puts you, if you just do the math in terms of future revenues, at around a $2 million price tag, that you are already looking at future revenues well north of half a billion dollars and getting closer to the billion-dollar mark, which makes it a blockbuster. I think that tailwind of pediatric expansion is only going to add to the number of patients that are being initiated per quarter because with kids, you have more children's hospitals at play, and they are used to these kinds of procedures, so they are going to be more productive as well as there is a more bigger addressable population.

Not to mention the fact that I think with these diseases, you want to treat these kids earlier and earlier. The sooner you treat them, the better because you don't have any organ damage or any permanent damage that will be caused by these diseases. All in all, I think a very favorable dynamic for CASGEVY over the coming quarters.

Yeah, at this point, most of the bottlenecks have been worked out by our partner, Vertex. I think initially when we started the launch, there was a lot of work required to get the reimbursement set up fully to be well-oiled in terms of prior authorizations, getting all the approvals, and mind you, we had to do this not just in the U.S., but several jurisdictions around the world. But good news is we're very strong payer coverage. Second thing was to get each of the hospitals contracted and you have individual requirements per hospital, potentially based on their own IT systems and what they needed to get these patients into flow. So that's all been done. Then the last thing is manufacturing capacity, and we're steadily increasing the capacity as we see more and more demand coming to be able to serve the needs of all these patients.

I would say at this point, most of those elements of friction have been dealt with and we're just now awaiting the patients to come into the funnel, move through the funnel, and ultimately get treated. It still does take over six months, potentially nine months plus to get a patient from initiation to treatment, but at some point, it's going to stop mattering because it's just a matter of getting into the funnel and then getting through the funnel and getting treated. All in all, I think that's looking very good and could be the basis for steady growth for a long period of time from here on out.

Yeah, I think we'll do everything we can, but it's not the manufacturing that's the major contributor to the time. I think the actual manufacturing itself happens pretty quickly. But a lot of patients opt to do exchange transfusions, for instance, so you have 2 to 3 months where someone's doing exchange transfusion before they even collected. Then after the drug is manufactured, sometimes people wait because they have to get their life in order, or they have to take a break, or they need vacation from work, et cetera. And so you do see some wait time there anyway. So it's never going to be one month. But at the same time, it doesn't matter at this point.