CRISPR Therapeutics Reports Phase 1 Zugo-Cel Data Showing Responses Across Autoimmune Diseases

CRISPR Therapeutics (NASDAQ:CRSP) has reported updated results from its ongoing Phase 1 clinical trial of zugocabtagene geleucel (zugo-cel), an investigational allogeneic CAR-T cell therapy targeting CD19, in patients with refractory autoimmune diseases.

As of October 15, 2026, 21 patients had received treatment across three disease groups: systemic lupus erythematosus (SLE), systemic sclerosis (SSc) and idiopathic inflammatory myopathy (IIM).

The company presented findings from the systemic sclerosis cohort at the American College of Rheumatology Convergence 2026 meeting.

Clinical Responses Reported Across Three Patient Groups

In the systemic sclerosis cohort, 83% of patients (five of six) who received standard lymphodepletion achieved a clinical response at Month 3. All five maintained their responses at the latest assessment, including four patients who had completed at least six months of follow-up.

Among patients with idiopathic inflammatory myopathy, all nine treated participants achieved either a moderate or major response based on the Total Improvement Score.

In the systemic lupus erythematosus cohort, two of three evaluable patients maintained remission without medication through at least Month 9, including one patient whose remission extended beyond one year.

The findings represent early clinical observations from a small patient population, with additional follow-up required to assess response durability.

Safety Data and Dose Escalation

CRISPR Therapeutics reported no dose-limiting toxicities among the 21 patients treated to date.

The company also reported no cytokine release syndrome above Grade 2, immune effector cell-associated neurotoxicity syndrome, graft-versus-host disease or deaths.

Dose escalation remains ongoing. Six patients treated at the 200 million CAR-positive cell dose demonstrated greater CAR-T cell expansion than patients receiving the 100 million CAR-positive cell dose.

The company has not yet established the optimal dose or long-term safety profile of zugo-cel.

Development Programme Targets Multiple Diseases

Naimish Patel, Chief Medical Officer of CRISPR Therapeutics, commented on the treatment needs of patients with refractory autoimmune conditions.

“Patients living with refractory autoimmune diseases such as systemic sclerosis, myositis and lupus often face progressive disease and a lifelong burden of immunosuppressive treatment,” Patel said.

Zugo-cel is an allogeneic therapy developed using donor-derived cells rather than cells collected individually from each patient. It does not require human leukocyte antigen (HLA) matching.

In addition to the rheumatologic autoimmune disease trial, CRISPR Therapeutics is evaluating zugo-cel in separate Phase 1 studies involving autoimmune hematologic and neurologic diseases, as well as relapsed or refractory B-cell malignancies.

The therapy remains investigational, and the ongoing studies will provide further information about its safety, dosing and clinical activity.

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