Express CRISPR gene editing and improvement, innovative CAR-T therapy was recognized by the FDA as an advanced therapy for regenerative medicine

▎Editor of WuXi AppTec's content team On November 22, 2021, CRISPR Therapeutics announced that the U.
S.
FDA has granted CTX110 Regenerative Medicine Advanced Therapy (RMAT) designation
.

CTX110 is an allogeneic CAR-T cell therapy that uses CRISPR gene editing technology to treat patients with CD19-positive B-cell malignancies
.

CTX110 locates the chimeric antigen receptor (CAR) targeting CD19 antigen into the cell's natural T cell receptor (TCR) site, and prevents the expression of endogenous TCR while introducing the CAR, thereby preventing graft-versus-host The occurrence of disease (GvHD)
.

At the same time, it also uses gene editing to knock out the expression of type 1 major histocompatibility complex, reducing the patient's immune system's attack on the imported cell therapy
.

▲Introduction of CTX110 (picture source: CRISPR Therapeutics company official website) The current multi-center, open-label phase 1 clinical trial is currently underway to evaluate the safety and effectiveness of CTX110 at multiple dose levels in the treatment of relapsed/refractory B-cell malignancies Sex
.

Previously obtained positive data showed that in patients with large B-cell lymphoma treated with CTX110 at a dose of more than 10^8 cells, the overall response rate based on the intention-to-treat analysis was 58%, and the complete response rate was 38%
.

After 6 months of treatment, the complete remission rate was 21%, and the longest remission lasted more than 18 months, showing the potential of CTX110 to bring sustained remission
.

In terms of safety, CTX110 shows differentiated positive safety features
.

The detailed remission information of each different dose is shown in the figure below: ▲Efficacy data of the phase 1 clinical trial of CTX110 (picture source: reference [2]) "The FDA grants RMAT based on encouraging clinical data, which is an important milestone.
, Means the transformative potential of CTX110 in the treatment of hematological malignancies
.

" Dr.
Samarth Kulkarni, CEO of CRISPR Therapeutics said, "We look forward to working closely with the FDA to bring this important new treatment to patients
.

"Reference: [1] CRISPR Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX110™ for the Treatment of Relapsed or Refractory CD19+ B-cell malignancies.
Retrieved November 22, 2021, from https://crisprtx.
gcs -web.
com/news-releases/news-release-details/crispr-therapeutics-announces-fda-regenerative-medicine-advanced[2] 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:// its-Phase-1-CARBON-Trial-of-CTX110-in-Relapsed-or-Refractory-CD19-B-cell-malignancies.
htmlDisclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.

This article is only For the purpose of information exchange, the views in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.

This article is not a treatment recommendation either
.

If you need guidance on treatment plans, please go to a regular hospital for treatment
.