Remove Engineering Remove RNA Remove Small Molecule
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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.

RNA 52
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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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Navigating the partnering galaxy

Dark Matter Blog

On the other hand, if you have a platform designed to be an engine for a standalone enterprise, capable of generating dozens of future drugs, then you might plot a course that brings you into the close orbit of multiple complementary partners. We all share a common understanding of the benefits and challenges of small molecule therapeutics.

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Johns Hopkins Researchers Identify CRISPR Dimmer

The Pharma Data

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

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Driving Toward Nanopores

Codon

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. 3 Nanopore engineers decided to cleverly exploit the natural speed limit of DNA polymerase to slow down DNA translocation through the pores.

DNA 96
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Cancer metastasis: breakthrough in therapeutic strategies

Drug Target Review

In the top row, human lung cancer cells were engineered to over-express GRP78 in the nucleus. Researchers from the Keck School of Medicine of USC used imaging techniques to study how the protein GRP78 controls cancer cell behavior. In the bottom row, cells lacked GRP78 in the nucleus.

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Targeting a human protein may stop Ebola virus in its tracks

The Pharma Data

Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase. But targeting Ebola virus polymerase has proven tough. To our knowledge, this is the first time GSPT1 has been linked to viral infection.”

Virus 52