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RNA processing in health and disease: challenges and opportunities of the field

Drug Target Review

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing.

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Molecules on the Move: Reversible BTKi in MS, THR-beta Agonist in NASH, and More

Drug Hunter

This article compiles recent high-profile clinical readouts and related news with small molecules of general interest and structures where they are available. Unlike sapropterin, sepiapterin is more active in cells and is also brain-penetrant.

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Reaching cruising altitude: New discovery tools to target RNA

Dark Matter Blog

The majority of small molecule drugs induce their therapeutic effects by seeking out and binding to their intended target while avoiding most other molecules in the dense milieu of the cell interior. Our overall mission at Arrakis is to expand the set of “druggable” targets for small-molecule medicines to include RNA.

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Women in STEM with Juliet Williams

Drug Target Review

This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.

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The rising impact of biomarkers in early clinical development

Drug Target Review

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

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New image-based cellular profiling tool peers deeply into metabolic biology

Broad Institute

The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds. That teaches us far more than looking for just a single informative data point.” Typically, studying drug therapies is an expensive and time-consuming undertaking.

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The Dangers of Mirrored Life

Codon

” The report accompanies an article in Science , also released today, entitled “Confronting Risks of Mirror Life.” For the article in Science , more than 30 scientists from ten countries are calling on the broader community to confront the risks of mirrored life. ” But what, exactly, is a mirrored organism?

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