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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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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. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers. billion in funding.

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Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

In-Vivo 162
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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Leading innovators in mammalian expression vectors for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry Mammalian expression vectors is a key innovation area in pharmaceutical Mammalian expression vectors are used to introduce a specific fragment of DNA into mammalian systems for RNA or protein expression. These systems include human and mouse cell lines, and even in vivo in live animals.

In-Vivo 130
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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

XTalks

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

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Leading innovators in gene splicing using nucleases for the pharmaceutical industry

Pharmaceutical Technology

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.