CRISPR Therapeutics Reports Strong 90% Response Rate and Lupus Remission in Landmark Zugo-Cel Update

CRISPR Therapeutics (NASDAQ:CRSP) is one of the best emerging technology stocks to invest in. On December 22, CRISPR Therapeutics released a comprehensive clinical update on zugocaptagene geleucel (zugo-cel), formerly known as CTX112. This investigational allogeneic (off-the-shelf) CAR T therapy targets CD19 and is currently being evaluated for both autoimmune diseases and hematologic malignancies. By using CRISPR Cas9 for targeted gene modification, zugo-cel is designed to enhance T-cell potency and evade immune detection without the need for HLA matching.

Preliminary data from an ongoing Phase 1 basket trial involving systemic lupus erythematosus/SLE, systemic sclerosis, and inflammatory myositis show that zugo-cel is well-tolerated and highly effective. As of the December 17 data cut-off, four patients (two with SLE and two with immune-mediated necrotizing myopathy) received a dose of 100 million cells. All four demonstrated rapid and deep peripheral B-cell depletion within 48 hours, which was sustained for at least 28 days. Notably, the first SLE patient treated has achieved drug-free DORIS clinical remission through Month 6. Following these results, the company has initiated an additional Phase 1 study for immune thrombocytopenic purpura and warm autoimmune hemolytic anemia.

In the oncology sector, zugo-cel has shown strong single-agent activity in patients with relapsed or refractory large B-cell lymphoma. At the recommended Phase 2 dose of 600 million cells, data from November 20 revealed an overall response rate of 90% and a complete response rate of 70% among ten patients. Long-term durability is also evident, with 67% of patients who reached the one-year mark remaining in complete response. The treatment demonstrated a four-fold higher cell expansion at the 600 million dose compared to the 300 million dose, maintaining a manageable safety profile with Grade 3 cytokine release syndrome occurring in only 17% of patients.

CRISPR Therapeutics (NASDAQ:CRSP) is a gene editing company that develops gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9 (CRISPR/Cas9, respectively) platform.

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Disclosure: None. This article is originally published at Insider Monkey.