CRISPR Therapeutics says CASGEVY momentum is building as zugo-cel and other pipeline data near

CRISPR Therapeutics said it is entering a “second phase” as CASGEVY moves beyond the initial launch and multiple pipeline readouts approach over the next 12 to 18 months. The company said CASGEVY’s commercial rollout is “gaining a lot of momentum,” while executives highlighted progress for zugo-cel, an allogeneic CAR-T being developed in oncology and autoimmune disease.

The company said its first 11 years were centered on developing CASGEVY for sickle cell disease and beta thalassemia and bringing the therapy to patients. With that program now commercialized through partner Vertex, it said more attention is shifting to a broader portfolio that includes cardiovascular, autoimmune, oncology and rare disease programs. The company said six assets are expected to generate data in the next 12 to 18 months: CTX310, zugo-cel, CTX611, CTX340, an Lp(a) program and an alpha-1 antitrypsin rare disease program.

On CASGEVY, the company said Vertex initiated about 100 patients in 2024, more than 300 patients in 2025 and has now initiated more than 500 patients. It said there can be a lag of two to three quarters from initiation to revenue recognition, and that CASGEVY generated $43 million in the first quarter. The current U.S. label covers patients ages 12 and older, and the company said it has submitted for an expansion to patients ages 5 and older. It also pointed to a reimbursement agreement in Germany and said a pediatric expansion could bring more children’s hospitals into the treatment network.

In oncology, the company described zugo-cel as its primary current focus and a next-generation CD19-targeted allogeneic CAR-T following CTX110, using healthy donor-derived cells manufactured at its facility in Framingham, Massachusetts. Executives said the goal is to approach “autologous-like efficacy” while improving safety and accessibility.

The company detailed a multi-edit engineering strategy for zugo-cel. The CAR is inserted into the T-cell receptor locus, which it said both knocks down TCR to reduce graft-versus-host disease risk and places CAR expression under endogenous regulatory control. An edit in the beta-2 microglobulin locus reduces MHC Class I presentation, which it said can lessen recognition and clearance by the host immune system and extend persistence. Two additional edits were described as designed to reduce exhaustion and improve functional activity during the persistence window.

The company said its studies to date have not required HLA matching and that it has not observed a correlation between HLA and cell expansion. It also said some patients have been redosed to deepen responses after partial response, with no evidence to date of an immune response preventing redosing. On lymphodepletion, the company said it uses standard chemotherapy lymphodepletion regimens similar to those used with autologous CAR-T in oncology, and that dose-for-dose, zugo-cel shows significantly greater expansion than CTX110.

The company said it is pursuing zugo-cel in both autoimmune disease and oncology in parallel, with the scale and direction of investment expected to be shaped by regulatory discussions. It said it hopes to engage regulators by year-end to determine whether single-arm registrational trials may be feasible in both settings or whether randomized studies would be required, particularly in oncology.

In autoimmune disease, the company said allogeneic CAR-T could be a “winner” due to scalability, a lower cost of goods under $10,000 and not needing to stop background therapy to harvest a patient’s T cells. It described an ongoing rheumatology basket trial in systemic lupus erythematosus, inflammatory myositis and scleroderma, with active recruitment across all three, and said it has reported two systemic lupus erythematosus patients achieving zero disease activity, including one patient with follow-up extending to nine months off background therapy.

For cardiovascular programs, the company said it presented data last year for CTX310 showing reductions of approximately 50% in LDL cholesterol or triglycerides after treatment. It described the early safety profile as favorable, with limited and self-resolving liver enzyme elevations observed. On CTX340, which targets angiotensinogen for hypertension, the company said reducing systolic blood pressure by 10 to 15 millimeters of mercury could be clinically meaningful.