TS-004607 — Using CRISPR/Cas9 genome editing can eliminate an inhibitory checkpoint receptor called TIGIT, which improves anti-tumor activity and cellular therapy for cancer. Chimeric antigen receptor technology also introduces an immune activating CAR within the TIGIT gene locus.
This IP is a gene therapy designed to treat cystic fibrosis; a fatal genetic disorder caused by CFTR mutations. Due to the large size of the gene, CF gene therapy efforts are limited. The first class of variants involved in this gene therapy improves stability and the second class of variants improv…