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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. The two patients treated with 0.45mg/kg had a time-averaged blood PCSK9 protein reduction of 59% and 84%.

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

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

In-Vivo 52
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Immunotherapy partnership advances space medicine research

Drug Discovery World

US company Vaxxinity and the University of Central Florida (UCF) have announced plans to work together to advance space medicine research. The research, funded by a grant from the State of Florida, aims to develop Vaxxinity’s active immunotherapies to prevent and mitigate muscle and bone wasting.

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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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Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. Merck will also make royalty payments on any approved products developed out of the partnership.

RNA 147
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First observation of the early link between proteins linked to Alzheimer’s disease

Scienmag

These results will help researchers to identify at an early stage the people most at risk of developing the disease, well before the first cognitive symptoms appear Credit: ©ULiège/CRC In Vivo Imaging Study conducted by researchers from the GIGA CRC In vivo Imaging laboratory at ULiège demonstrates, for the first time in humans, how the […]. (..)

In-Vivo 52
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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