CRISPR Therapeutics presented preliminary data from its ongoing Phase 1/2 clinical trial evaluating CTX112, a next-generation CD19 allogeneic CAR T cell therapy, in relapsed or refractory (R/R) CD19-positive B-cell malignancies at the 2024 American Society of Hematology (ASH) Annual Meeting. The data demonstrated that CTX112 has the potential to provide meaningful clinical benefit with a well-tolerated safety profile using a standard lymphodepletion protocol. This suggests a promising therapeutic option for heavily pre-treated patients.
The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX112 for the treatment of R/R follicular lymphoma and marginal zone lymphoma. This designation is designed to expedite the drug development and review processes for promising regenerative medicine products, offering benefits such as intensive FDA guidance and potential priority review. The RMAT designation underscores the FDA's recognition of CTX112's potential to address unmet medical needs.
The Phase 1/2 study included data from 12 subjects treated across dose levels ranging from 30 x 10^6 to 600 x 10^6 CAR+ T cells, showing responses at all dose levels. The safety profile was well tolerated across all dose levels, with no adverse events of interest detailed in the preliminary data. These findings support the potential of allogeneic cell therapies to offer an off-the-shelf treatment option for patients with B-cell malignancies.
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