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Reinventing the small molecule toolbox: from proteins to RNA

Dark Matter Blog

Prior to 2015, I had a casual relationship, at best, with targeting RNA. The bulk of my nearly three decades of experience up to that point was with drugging protein targets using a variety of modalities, but principally small molecules. But do those hits bind the same structures in the wild?

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A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. Dramatic discoveries of small molecules targeting SMN2 Prior to those outstanding therapeutic achievements, in 2015, a group from Novartis published an important paper in Nature Chemical Biology.

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Lead Pharma Teams with Roche on Oral Small Molecules for Immune-Mediated Diseases

The Pharma Data

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. .

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The power of combinations in blood cancers

Drug Target Review

These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers. 2015);126(1):9-16. 2015);126(4):454-462. Clonal hematopoiesis of indeterminate potential and its distinction from myelodysplastic syndromes.

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Antibody-drug conjugates payloads: then, now and next

Drug Target Review

Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 6 Combining the effect of payloads with different mechanisms of action – an approach that revolutionised small molecule chemotherapy – also holds the promise of enhanced therapeutic activity for ADCs.

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Fearlessly Forward to our Destination

Dark Matter Blog

Our terra firma is small-molecule medicines. As we know, small molecules have been the mainstay of our pharmacopeia for several centuries and have, almost without exception, targeted proteins. This is why Arrakis set out in 2015 on an expedition to figure out how to develop small molecules that can target RNA.

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Got It Down Cold: Cryo-Electron Microscopy Named Method of the Year

NIH Director's Blog: Drug Discovery

Very impressive, and just one of the many reasons why the journal Nature Methods recently named cryo-EM its “Method of the Year” for 2015 [2]. Cryo-EM maps showing the contacts between small molecules and proteins can help to explore questions such as why one drug is better than another or why certain drugs fail. Nature Methods.