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FDA sets out advice to developers of gene editing medicines

Bio Pharma Dive

Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing.

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Regeneron expands in gene editing with Mammoth deal

Bio Pharma Dive

“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.

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Intellia, with first results, delivers a 'landmark' for CRISPR gene editing

Bio Pharma Dive

Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.

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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene-editing firm Prime Medicine files $140m public offer

pharmaphorum

Gene-editing biotech Prime Medicine has priced its IPO, hoping to raise $140m to advance its pipeline of one-shot therapies for severe genetic disorders.

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Prime Medicine Gets FDA Okay for First Trial of Gene Editing Technique

BioSpace

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.

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”