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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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Talem and Libera Bio partner for discovery of AI-driven antibodies

Pharmaceutical Technology

The partnership will use nanotechnology, which was proven in vivo, for providing antibodies inside tumour cells and provides a potential solution to address the medical challenge. Under the collaboration, Libera Bio and Talem Therapeutics will together develop new antibodies for use with MPN delivery.

Antibody 262
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This week in drug discovery (18-22 March) 

Drug Discovery World

The top stories: ‘Dual-target’ CAR-T cell therapy shrinks brain tumours Targeting two brain tumour-associated proteins – rather than one – with CAR-T cell therapy has shown promise as a strategy for reducing solid tumour growth in patients with recurrent glioblastoma (GBM).

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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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Partnership to accelerate neuroscience drug discovery  

Drug Discovery World

ACROBiosystems, through ACRO Certify and under its Aneuro brand, has launched in vivo electrophysiology solutions for neuroscience research with Diagnostic Biochips. Together, the two companies are committed to a collaborative effort to accelerate neurological drug discovery and commercialisation.

In-Vivo 52
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AI company raises $3m to develop antibodies targeting GPCRs

Drug Discovery World

Antiverse has successfully identified functional antibody clusters targeting two G-protein-coupled receptors (GPCRs), both displaying versatile binding profiles and nanomolar affinity. The company has raised $3 million in funding, which will enable in-house development of the antibodies showing the highest affinity blocking function.