Gene editing

Results from a Phase I study presented Friday at the ASGCT 2024 annual meeting showed that despite a good safety profile, Excision BioTherapeutics’ HIV gene editor failed to suppress viral activity.
The biotech touted its prime editing technology at ASGCT on Tuesday after receiving FDA clearance last week for a clinical study of a drug candidate based on the platform.
Cell and gene therapy company Cellectis announced Monday it has completed a $140 million investment from AstraZeneca that extends the biotech’s cash runway into 2026.
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.
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