Gene editing

A pair of biotechs burst onto the scene Tuesday, exiting stealth with $100 million in financing each and tapping executives who made their names at bluebird bio and Karuna Therapeutics.
Verve Therapeutics is pausing enrollment in the Phase Ib Heart-1 study for its gene editor VERVE-101 after a patient developed grade 3 laboratory abnormalities, the company announced Tuesday.
FDA
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.
The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.
Allogene Therapeutics and Arbor Biotechnologies will use their allogeneic CAR T and next-generation gene-editing platforms to develop novel off-the-shelf CAR-T therapies for autoimmune diseases.
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
Separate challenges exist for companies developing gene therapies for rare and common cardiovascular conditions, experts told BioSpace.
From entering college in China at just 15 to co-inventing a drug for Bayer, Mei’s career journey has been far from average. Now CMO at Editas, he’s not done yet.
CRISPR gene-editing has had its first ever approval in the UK. Will the FDA follow suit? What can patients expect the price tag to be?
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