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

Drug Discovery World

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. 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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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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News from AACR 2024: Sunday’s highlights

Drug Discovery World

Aviv Regev, Head and Executive Vice President of Research and Early Development at Genentech, opened the session with a discussion of how advances in genomic sequencing are fostering the development of data-rich cell atlases and what that data could mean for new insights into cancer biology and novel therapeutics.

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. 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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Conversations from ESMO Targeted Anticancer Therapies Congress 2024 

Drug Discovery World

More importantly, it also highlighted that significant challenges remain in selecting the right therapies for the right patients despite the progress made through genomic testing and implementation of circulating tumour (ct) DNA analyses. We were there to present the latest findings from our research.

RNA 64
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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