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Intellia cleared to start key CRISPR drug study in UK

Bio Pharma Dive

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition. Below, we discuss some of these challenges in cell therapy trials.

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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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Verve starts trials of cholesterol drug in test of base editing technique

pharmaphorum

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Generating Over a Billion Cells with CRISPR for Next Generation Cell Therapies

XTalks

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

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CRISPR pioneers Doudna and Charpentier claim Nobel chemistry prize

pharmaphorum

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. The technique introduces a break in a specific place within DNA that triggers a self-repair mechanism. million) Nobel Prize award. “In

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Could CRISPR cure HIV? US biotech Excision raises $60m to find out

pharmaphorum

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. Excision wants to take its lead therapy candidate, EBT-101, into a phase 1/2 clinical trial in patients with chronic HIV infection with the proceeds from this latest financing round.