Articles related to GENE THERAPY

A quartet of current clinical trials involving first-in-class molecules and CRISPR gene editing show great promise. STELLAR investigates a fusion protein in PAH, CodeBreak 200 studies a first-in-class of KRAS inhibitor, EMERGENT-2 focuses on a new treatment for schizophrenia, and NCT04601051 studies a gene editing drug for one form of amyloidosis.

In the study,155 FDA-approved drugs with 136 potentially targetable genes were identified.One-hundred forty-six (94%) of the drugs showed no or low genetically predicted drug response. Although ATC-carrying BRAF mutations can benefit from BRAF inhibitors and this effect might be enhanced by a combined strategy including PIK3CA inhibitors in some of the patients, alterations in BRAFWT ATC are not directly targeted by currently FDA-approved options.

Targeted therapy gene mutations may: (a) encode a protein that can be targeted in a manner distinct from the WT protein; (b) cause abnormal activation of a protein that is druggable but for which mutant-specific targeting has not been achieved; or (c) create novel molecular dependencies that are druggable. This article describe key examples of genetic drivers and associated modes of druggability, considers the clinical and research challenges in the field, and discusses new approaches to maximizing therapeutic benefit of targeted therapies.

Blocking expression of FOXK2 in cancer stem cells inhibited growth of these cells, reducing their so-called “stemness,” making them more like normal cells.

Adagrasib 600 mg twice a day was well tolerated and exhibited antitumor activity in patients with advanced solid tumors harboring the KRASG12C mutation.

The NR2F1 gene normally prevents pre-malignant cells from spreading to other parts of the body. HER2 suppresses NR2F1, allowing pre-cancerous cells to move to other organs where they can become cancerous.