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Single-course in vivo base editing therapy proven to lower cholesterol

Drug Discovery World

The trial participants have heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterised by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

In-Vivo 52
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FDA clears in vivo gene editing therapy for trials in US

Drug Discovery World

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo 52
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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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Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Roots Analysis

The global genome editing market is anticipated to grow at a CAGR of 12.6% The global genome editing market is anticipated to grow at a CAGR of 12.6% Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. during the forecast period 2023-2035.

Genome 40
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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 93
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Delivering on the promise of gene editing

Drug Discovery World

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions. In one example, Choi et al.

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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.