CRISPR Therapeutics' CRSP Provides Significant Updates on Zugocaptagene Geleucel in Autoimmune Diseases and Hematologic Malignancies
Date: December 22, 2025
Source: GlobeNewsWire
Overview of CRISPR Therapeutics' Developments
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company dedicated to pioneering gene-based therapies, has released new insights into its investigational treatment, zugocaptagene geleucel (zugo-cel), which targets CD19. The updates pertain to its applications in autoimmune disorders and hematologic malignancies.
Promising Results in Autoimmune Disease Trials
Recent Phase 1 clinical trials involving zugo-cel have revealed compelling preliminary data. Four patients suffering from autoimmune diseases exhibited deep B-cell depletion that was sustained for at least 28 days. Notably, a patient with systemic lupus erythematosus (SLE) achieved Definitions of Remission in SLE (DORIS) remission by Month 6 following treatment at a dose of 100 million cells.
- Deep B-cell Depletion: Observed within 1-2 days, maintained over the first month.
- Clinical Improvements: All patients involved in the study showed significant clinical progress at the Day 28 assessment.
- Well Tolerated: No instances of high-grade cytokine release syndrome (CRS) or neurotoxicity were reported.
Expansion of Clinical Trials
In addition to the encouraging data for autoimmune diseases, CRISPR Therapeutics has initiated a new Phase 1 basket trial investigating zugo-cel in patients with refractory primary immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA).
Chief Medical Officer Naimish Patel, M.D., expressed optimism: “Preliminary data from zugo-cel in patients with rheumatologic autoimmune diseases have been encouraging, and the therapy has been well tolerated to date.”
Impressive Efficacy in Hematologic Malignancies
The clinical experience with zugo-cel in hematologic malignancies, particularly in relapsed or refractory (R/R) large B-cell lymphoma (LBCL) patients, has shown promising results:
- Overall Response Rate (ORR): 90% (9 out of 10 patients)
- Complete Response Rate (CRR): 70% (7 out of 10 patients)
- Long-term Remission: 67% (2 out of 3 patients) remained in complete response after 12 months.
The recommended Phase 2 dose for further trials in LBCL has been established at 600 million cells.
Collaboration with Lilly for Enhanced Treatment Options
CRISPR Therapeutics has also announced a collaborative agreement with Lilly to further investigate the combination of zugo-cel with pirtobrutinib in treating aggressive B-cell lymphomas. This partnership highlights the significant potential for zugo-cel in expanding treatment strategies within oncology.
Future Outlook
Looking ahead, CRISPR Therapeutics anticipates providing additional updates on zugo-cel in the second half of 2026 across both autoimmune diseases and hematological malignancies.
About Zugocaptagene Geleucel (zugo-cel)
Zugocaptagene geleucel (zugo-cel) is an allogeneic CAR T cell therapy designed specifically for targeting CD19. It employs CRISPR technology for targeted gene editing and does not require HLA matching, making it a versatile treatment option for various autoimmune and oncological conditions.
About CRISPR Therapeutics
Established over a decade ago, CRISPR Therapeutics is a leader in gene editing, focused on developing transformative therapies to address serious diseases. The company recently celebrated a significant milestone with the approval of CASGEVY®, marking the world's first CRISPR-based treatment approved for managing sickle cell disease and transfusion-dependent beta thalassemia.