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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

In-Vivo 105
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Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Zinc fingers. billion in funding.

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Another Milestone: LogicBio Successfully Edits Genes in Children

BioSpace

On Monday, LogicBio Therapeutics unveiled clinical trial results demonstrating the first-ever in vivo, nuclease-free genome editing in little humans.

In-Vivo 82
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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52
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Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis

pharmaphorum

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). Precision Bio aims to address those challenges using its proprietary ARCUS nuclease platform, which is designed to insert a transgene with high accuracy into the genome.

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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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In the News: October Regulatory and Development Updates

Camargo

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. of hyponatremia, or low blood sodium levels. In October, the U.S.