CRISPR Therapeutics AG (CRSP) shared some genuinely encouraging updates Monday on zugocaptagene geleucel, or zugo-cel for those who prefer not to twist their tongue into knots. The investigational allogeneic CAR T therapy targeting CD19 is being developed for both autoimmune diseases and blood cancers, and the early data suggests it might actually work pretty well at both.
The Autoimmune Story
Zugo-cel is currently enrolled in an ongoing Phase 1 basket trial for autoimmune rheumatologic diseases. The preliminary results have been encouraging, with the therapy demonstrating good tolerability so far.
As of the December 17 data cutoff, four patients received a 100 million cell dose and were followed for at least 28 days post-infusion. Here's what happened: the therapy's cell expansion matched what researchers observed at the same dose in the ongoing B-cell lymphoma trial. Patients experienced rapid and deep B-cell depletion in the periphery within the first one to two days, which was maintained throughout the first month of treatment. The repopulating B-cells showed a shift toward an unswitched, naïve repertoire.
All four patients demonstrated significant clinical improvement at the Day 28 assessment. On the safety front, treatment was well-tolerated with no high-grade cytokine release syndrome (CRS) or immune-effector cell-associated neurotoxicity syndrome (ICANS) observed.
Clinical trials across autoimmune indications remain ongoing, with the company planning additional updates in the second half of 2026. CRISPR Therapeutics has also initiated a Phase 1 trial evaluating zugo-cel in immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA).
The Cancer Data Gets Interesting
The immuno-oncology results are where things get particularly compelling. Zugo-cel was administered following a standard lymphodepletion regimen with fludarabine and cyclophosphamide. A total of 39 patients have been treated across four dose levels, with the recommended Phase 2 dose recently established at 600 million cells for the large B-cell lymphoma (LBCL) cohort.
As of the November 20, 2025 data cutoff, 10 patients with relapsed/refractory LBCL received the 600 million cell dose and had at least one month of follow-up. The results: an overall response rate of 90% (nine out of 10 patients) and a complete response rate of 70% (seven out of 10). Notably, one patient who had relapsed following autologous CAR T cell therapy achieved a complete response.
Among patients who completed 12 months of follow-up, 67% (two out of three) remained in complete response at the 12-month evaluation. Peak mean CAR T cell expansion reached approximately 1,700 cells per microliter at the recommended Phase 2 dose, representing roughly a four-fold higher expansion compared with patients receiving 300 million cells.
The safety profile was manageable. Among all LBCL patients treated at the recommended Phase 2 dose (12 patients total), rates of Grade 3 CRS, ICANS, and serious infections were 17%, 17%, and 8%, respectively. Importantly, no Grade 3 ICANS or CRS was observed at the 100 million cell dose currently being studied in autoimmune basket trials.
The Phase 1/2 trial in relapsed/refractory B-cell malignancies continues, with the company expecting to provide additional updates in the second half of 2026.
Expanding The Program
CRISPR Therapeutics has established a collaboration and clinical supply agreement with Eli Lilly and Co (LLY) to evaluate zugo-cel together with pirtobrutinib in aggressive B-cell lymphomas, further expanding the program's oncology development.
CRISPR Therapeutics shares were up 3.46% at $57.79 at the time of publication on Monday.