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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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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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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. They wanted someone who had lots of experience in drug development, was a molecular biologist, and was stubborn enough to take on CRISPR!” Zinc fingers. billion in funding.

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Artificial Intelligence innovation: Leading companies in in-silico drug discovery

Pharmaceutical Technology

DNA binding site prediction, peptide structure optimisation, and AI-assisted genome analysis are some of the accelerating innovation areas, where adoption has been steadily increasing. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189
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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

Single cell RNA sequencing technology has been extensively applied to understand heterogeneity among tumor cells, within the tumor microenvironment and during cell development. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. The Power of Single Cell Technology.

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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver.