Express gene editing to create "ready-to-use" CAR-T therapy, phase 1 clinical trial results are positive

▎The content team editor of WuXi AppTec today, CRISPR Therapeutics announced that the allogeneic CAR-T cell therapy CTX110, modified with CRISPR gene editing technology, has achieved positive safety and efficacy in a phase 1 clinical trial for the treatment of CD19-positive B-cell cancers Result
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Preliminary results show that in patients with large B-cell lymphoma, a higher dose of CTX110 single-dose treatment achieves a total remission rate of 58% and a complete remission rate of 38%
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CTX110 is an allogeneic CAR-T cell therapy manufactured using CRISPR gene editing technology
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It will target CD19 antigen chimeric antigen receptor (CAR) to knock into the cell's natural T cell receptor site, and prevent the expression of endogenous TCR while introducing CAR, thereby preventing graft-versus-host disease (GvHD).
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▲Introduction to CTX110 (picture source: CRISPR Therapeutics) In this clinical trial, a total of 26 large B-cell lymphoma patients received treatment with CTX110 and followed up for at least 28 days
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The test results showed that among patients treated with CTX110 at a dose of more than 10^8 cells, the total response rate based on the intention-to-treat analysis was 58%, and the complete response rate was 38%
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See the figure below for detailed relief information for each different dose
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▲CTX110 Phase 1 clinical trial efficacy data (picture source: Reference [1]) After receiving treatment for 6 months, the complete remission rate was 21%, and the longest remission lasted more than 18 months, showing that CTX110 brought Potential for sustained relief
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In terms of safety, CTX110 shows differentiated positive safety features
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There was no graft-versus-host disease, and all cytokine release syndromes (CRS) were grade 1 or 2
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In patients who received CTX110 re-dose, the frequency and severity of CRS did not increase
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Only one patient with HHV-6 encephalitis developed immune effector cell-related neurotoxicity syndrome (ICANS) above grade 3
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The specific safety features are as follows: Image source: Reference [1] "We are pleased to share the positive results of this clinical trial, which shows that CTX110 can provide patients with large B-cell lymphoma with a'ready-to-use' type that can be used immediately Therapies, which have the same efficacy as autologous therapy, and have differentiated safety characteristics
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" Dr.
Samarth Kulkarni, CEO of CRISPR Therapeutics, said, "Moreover, with the use of consolidation drug delivery, we have the potential to further improve the efficacy
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Based on these results , We plan to start a registered clinical trial in 2022
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"Reference: [1] CRISPR Therapeutics Reports Positive Results from its Phase 1 CARBON Trial of CTX110™ in Relapsed or Refractory CD19+ B-cell malignancies.
Retrieved October 12, 2021, from https:// release/2021/10/12/2312955/0/en/CRISPR-Therapeutics-Reports-Positive-Results-from-its-Phase-1-CARBON-Trial-of-CTX110-in-Relapsed-or-Refractory-CD19- B-cell-malignancies.
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