ARTICLE | Translation in Brief

A stem cell therapy for brain metastases in breast cancer; plus a nanoparticle gene editing delivery tech and more

BioCentury’s roundup of translational news

March 5, 2021 11:27 PM UTC

A team led by Khalid Shah from Harvard Medical School engineered allogeneic stem cells with an anti-EGFR camelid antibody fused to a death receptor ligand. Published in Science Advances, the therapy induced apoptosis of cancer cells and increased survival in mouse models of basal-like breast cancer with brain metastases. Amasa Therapeutics, a company developing stem cell–based therapies for cancer, was founded based on work by Shah’s team.

Lipid nanoparticle-mediated gene editing delivery tech
An article in PNAS described a lipid nanoparticle delivery platform carrying Cas9 mRNA and a guide RNA that mediated ANGPTL3 gene knockdown specifically in the liver of mice. The therapeutic effects, including reductions in serum ANGPTL3 protein, low density lipoprotein cholesterol and triglyceride levels, lasted at least 100 days after a single dose. The method, developed by Tufts University and the Broad Institute of MIT and Harvard researchers including Editas Medicine Inc. (NASDAQ:EDIT) co-founder Feng Zhang, adds to the growing list of gene editing delivery technologies...