CRISPR Therapeutics makes strategic shift

Days before expected FDA approval of its sickle cell gene therapy, Casgevy, CRISPR Therapeutics unveils revisions to its immuno-oncology portfolio and broadens its scope to include autoimmune disorders.

By: Gorm Palmgren - Dec. 5, 2023
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#CRISPRMED24

CRISPR Therapeutics is updating its immuno-oncology pipeline, with a key focus on next-generation CAR T cell candidates CTX112 and CTX131. However, in a significant development, the company is also expanding its trials into autoimmune diseases, particularly with CTX112, which is showing great promise in this regard. The news was shared in a press release yesterday.

CTX112 targets CD19 and is currently in clinical trials for B-cell malignancies, while CTX131 targets CD70 and is being tested for solid tumors. The gene edits in these candidates - Regnase-1 knockout and TGFBR2 knockout - enhance CAR T potency and reduce exhaustion. Patients previously treated with first-generation candidates CTX110 and CTX130 will transition to long-term follow-up.

What's noteworthy is that CRISPR Therapeutics plans to initiate new clinical trials of CTX112 in autoimmune indications. Early clinical studies have demonstrated that CD19-directed autologous CAR T therapy can lead to long-lasting remissions in various autoimmune conditions. CTX112 has the potential to offer similar results with added advantages, including scalability, cost-effectiveness, and eliminating the need for patient apheresis.

The company intends to kick off a clinical trial for systemic lupus erythematosus (SLE) in the first half of 2024, with plans to explore additional autoimmune indications in the future. This expansion highlights CRISPR Therapeutics' commitment to harnessing CAR T technology for treating autoimmune diseases alongside its immuno-oncology efforts.

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News: CRISPR Therapeutics makes strategic shift
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