Gene editing

Citing a strategic prioritization by Moderna, Metagenomi announced Wednesday that the partners have “mutually agreed” to terminate their gene editing agreement.
The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.
Regeneron will use Mammoth Biosciences’ tiny Cas enzymes to deliver in vivo CRISPR-based gene editing therapies to tissues and cell types beyond the liver, the companies announced Thursday.
From gene-corrected cell therapies to a new CAR-T, the cell and gene therapy space looks to expand its reach into the market.
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.
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