Accession PRJCA002488
Title Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small cell lung cancer
Relevance Medical
Data types Whole genome sequencing
Raw sequence reads
Genome sequencing
Organisms Homo sapiens
Description CRISPR-Cas9 system is an efficient genome editing tool but its clinical application continues to be controversial. This is a phase 1 trial on safety and feasibility of PD-1 edited T Cells via CRISPR-Cas9 on patients with advanced non-small cell lung cancer (NSCLC) after failing three or more lines of treatments. Total of 12 patients had sufficient CRISPR-Cas9 gene-edited T cells for clinical infusion and had completed the course of treatment for safety evaluation. The mean mutation frequency of off-target events was 0.06% (range 0.00-0.25%) at 18 candidate sites by next generation sequencing (NGS). All treatment-related adverse events (AEs) were grade 1/2. Gene-edited T cells and discernible T-cell receptor (TCR) diversity were detectable in peripheral blood at 8.0 to 52.0 weeks after infusion. Median progression-free survival (PFS) was 7.9 weeks (95% confidence interval [CI], 7.3 to not available [NA]) and median overall survival was 42.6 weeks (95% confidence interval [CI], 24.4 to NA). Clinical application of CRISPR-Cas9 gene-edited T cell is safe and feasible. Further investigation on clinical efficacy is warranted. (MHC-001 ClinicalTrials.gov number, NCT02793856)
Sample scope Multiisolate
Release date 2020-04-16
Publication
PubMed ID Article title Journal name DOI Year
32341578 Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer Nature Medicine 10.1038/s41591-020-0840-5 2020
Grants
Agency program Grant ID Grant title
National Science and Technology Major Project 2017ZX09304023
Submitter Su    Xiaoxing  (suxiaoxing_work@foxmail.com)
Organization BerryOncology
Submission date 2020-04-04

Project Data

Resource name Description