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

Pharmaceutical Technology

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 Artificial Intelligence in Pharmaceuticals: In-silico drug discovery. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189
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New drug could help overcome tumour resistance to treatment

Drug Discovery World

New study results have demonstrated the potential of a new drug to reprogramme the immune profile in the tumour microenvironment (TME) and convert resistant tumours to responders to Immune Checkpoint Blockage (ICB) therapies. The post New drug could help overcome tumour resistance to treatment appeared first on Drug Discovery World (DDW).

Drugs 98
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UK company identifies potential new treatment for lupus

Drug Discovery World

Domainex has revealed human ex vivo data indicating that DMXD-011 could be effective in treating interferonopathies such as lupus, Sjögren’s syndrome, and scleroderma. The drug candidate is an inhibitor of protein kinases TBK1 and IKK-epsilon, which previous studies suggest should be highly-effective in the treatment of inflammatory diseases.

In-Vivo 52