Tag Archive for: gene editing

Verve Therapeutics has suspended enrollment in the Phase Ib Heart-1 study evaluating its lead gene editing program VERVE-101 following a serious adverse event, the company announced Tuesday.

Moderna-backed Metagenomi is a precision genetics medicines company with what it describes as a comprehensive genome editing toolbox consisting of programmable nucleases, base editors, RNA-mediated integration systems and CRISPR-based techniques.

Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) said.

When given at potentially therapeutic doses, Verve’s base editor led to strong reductions in LDL cholesterol and PCSK9 levels in patients with heterozygous familial hypercholesterolemia.

Data show the potential of Editas’ sickle cell disease and transfusion-dependent beta thalassemia gene therapy candidate, but it might not be enough to overtake Vertex and CRISPR Therapeutics.

When you hear the phrase “gene therapy,” what comes to mind? A revolutionary cure for an exotic disease? A risky experimental approach that messes with nature? Something in between?

The Massachusetts-based company has claimed the year’s largest fundraise so far and secured a powerhouse R&D partner in Novo Nordisk to develop gene editing medicines.

AstraZeneca inked a non-exclusive licensing agreement with Revvity for access to the Massachusetts biotech’s proprietary gene editing technology to develop cell therapies for cancer and immune-mediated conditions, Revvity announced Thursday.

Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials. As these programs mature, 2023 could be a pivotal year for companies in the space.

Graphite Bio is discontinuing the development of nulabeglogene autogedtemcel (nula-cel), its lead asset, and shaving off about 50% of its workforce.