In one of the longest studies to date in high-risk heart disease, researchers found no significant difference in death rates over more than 10 years—as well as in cases of heart attack or stroke—between people that had received a drug-eluting stent for their blocked coronary artery compared with those who underwent standard bypass surgery.

Ipsen has terminated a pivotal trial of its troubled rare disease drug palovarotene in response to the ongoing clinical hold. The action likely deprives Ipsen of a shot at generating data to bring a drug it bought in a $1.3 billion (€1.2 billion) deal to market in a blockbuster indication.

The FDA warned patients, parents and providers that various EpiPen models could malfunction and spring their needles early.

Redpin Therapeutics has raised $15.5 million to bankroll chemogenetic R&D. The initial close of the series A positions Redpin to move a controllable gene therapy for the treatment of indications such as epilepsy and pain toward an IND.

Revealing yet another super-power in the skillful squid, scientists have discovered that squid massively edit their own genetic instructions not only within the nucleus of their neurons, but also within the axon — the long, slender neural projections that transmit electrical impulses to other neurons. This is the first time that edits to genetic information have been observed outside of the nucleus of an animal cell.

Chimeric antigen receptor (CAR) T cell therapy has been a game-changer for blood cancers but has faced challenges in targeting solid tumors. Now researchers from the Perelman School of Medicine at the University of Pennsylvania may have an alternative to T cell therapy that can overcome those challenges. Their research shows genetically engineering macrophages – an immune cell that eats invaders in the body – could be the key to unlocking cellular therapies that effectively target solid tumors. The team was able to show these CAR macrophages can kill tumors both in human samples in the lab and in mouse models. They published their proof-of-concept paper in Nature Biotechnology today.

Editing Multiple Genome Fragments At A Time

Drug therapies that help older adults maintain their skeletal muscle mass and physical function for longer could be a step closer after researchers at the University of Birmingham identify a key mechanism that drives the clearance of damaged mitochondria.

A new study has shown a novel peptide antagonist, given in combination with a PD-1 inhibitor, to be safe and well-tolerated in patients with advanced, refractory pancreatic and rectal cancer. The highest dose tested had a good safety profile and was recommended for use in future patient trials, as reported in Journal of Pancreatic Cancer, a peer-reviewed open access publication from Mary Ann Liebert, Inc., publishers.

Researchers have used high-throughput screening of adeno-associated viral (AAV) vector capsid libraries to maximize the likelihood of obtaining AAV variants with desired properties. As a result of these experiments, they gained some unexpected insights, reported in an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.