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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%.

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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 post Immunotherapy partnership advances space medicine research appeared first on Drug Discovery World (DDW).

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Start-up aims to advance transformative genetic medicines

Drug Discovery World

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).

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Oncotarget: A novel format for recombinant antibody-interleukin-2 fusion proteins

Scienmag

Oncotarget recently published a novel format for recombinant antibody-interleukin-2 fusion proteins exhibits superior tumor-targeting properties in vivo which reported that here, the authors describe four novel formats for the L19-IL2 fusion Credit: Correspondence to – Dario Neri – dario.neri@pharma.ethz.ch

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Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

Cas, also called CRISPR associated proteins, are a family of nucleases such as Cas9, Cas12a, Cas12b, and CasX that process these sequences. 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: CRISPR nucleases.

In-Vivo 162
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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 […]. (..)

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