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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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Clinical study first step towards treating respiratory syncytial virus 

Drug Discovery World

Enanta Pharmaceuticals, a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, has dosed the first subject in its Phase I clinical trial of EDP-323, a novel, oral L-protein inhibitor in development for the treatment of respiratory syncytial virus (RSV). . Context .

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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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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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Navigating the partnering galaxy

Dark Matter Blog

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.