CRISPR Therapeutics CRISPR Therapy Shows 90% Response Rate In Aggressive Blood Cancer

CRISPR Therapeutics AG (NASDAQ:CRSP) on Monday provided updates on zugocaptagene geleucel (zugo-cel, formerly CTX112), its investigational allogeneic CAR T targeting CD19, in development for autoimmune disease and hematologic malignancies.

Autoimmune Disease

Zugo-cel, targeting CD19, is in an ongoing Phase 1 basket trial in autoimmune rheumatologic diseases.

Preliminary clinical data from the Phase 1 study have been encouraging, and zugo-cel has been well-tolerated to date.

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As of the data cut-off on December 17, four patients have been treated at a dose of 100 million cells and followed for at least 28 days post-infusion:

Zugo-cel cell expansion is comparable to that observed at the same dose in patients in the ongoing B-cell lymphoma trial.

Rapid and deep B-cell depletion in the periphery was observed within the first 1-2 days and maintained over the first month of treatment, with repopulating B-cells demonstrating a shift toward an unswitched, naïve repertoire.

All patients demonstrated significant clinical improvement at the Day 28 assessment.

Treatment has been well-tolerated, with no high-grade cytokine release syndrome (CRS) or immune-effector cell-associated neurotoxicity syndrome (ICANS) observed.

Clinical trials in autoimmune disease remain ongoing across indications. The company expects to provide additional updates in the second half of 2026. In addition, a Phase 1 clinical trial for zugo-cel has been initiated in immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA).

Immuno-Oncology

Zugo-cel was administered after a standard course of lymphodepletion with fludarabine and cyclophosphamide.

A total of 39 patients have been treated across all four dose levels. The recommended Phase 2 dose (RP2D) was recently endorsed at the 600 million cell dose for the large B-cell lymphoma (LBCL) cohort.

As of the data cut-off of November 20, 2025, 10 patients with R/R LBCL have been treated at the RP2D of 600 million cell dose and have had at least one month of follow-up, with the following observations:

An overall response rate (ORR) of 90% (9/10) and a complete response rate (CRR) of 70% (7/10) were observed, including a complete response (CR) in a patient who relapsed following autologous CAR T cell therapy.

Among patients who have completed 12 months of follow-up, 67% (2/3) remained in CR at the 12-month evaluation.

Peak mean CAR T cell expansion of approximately 1,700 cells/µL was observed at the RP2D, representing approximately a four-fold higher expansion compared with patients receiving 300 million cells.

Rates of Grade 3 CRS, ICANS, and serious infections were 17%, 17%, and 8%, respectively, among all LBCL patients treated at the RP2D (n=12).

No Grade 3 ICANS or CRS has been observed at the 100 million cell dose, which is the dose currently being studied in the autoimmune basket trials.

The Phase 1/2 clinical trial in R/R B-cell malignancies is ongoing. The company expects to provide additional updates in the second half of 2026.

CRISPR Therapeutics has also established a new collaboration and clinical supply agreement with Eli Lilly and Co. (NYSE:LLY) to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program’s development in oncology.

CRSP Price Action: CRISPR Therapeutics shares were up 3.46% at $57.79 at the time of publication on Monday, according to Benzinga Pro data.

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