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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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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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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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To the Builders Blazing our Path to Amazing Medicines

Dark Matter Blog

Arrakis has gone through many significant transitions since its founding in 2015. Or should I say back to retirement, as he was comfortably relaxing at home back in 2016 when Jen Petter lured him back into the fray with the siren call of developing a new class of RNA-targeted small-molecule medicines here at Arrakis.

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Sosei Heptares scores $5m milestone payment from Pfizer

The Pharma Data

The collaboration has leveraged Sosei’s StaR technology and Structure-based Drug Design (SBDD) capabilities to design oral small molecules that modulate different G protein-coupled receptor (GPCR) targets across multiple disease areas. At the time, Pfizer made a $33m equity investment in Sosei as part of the collaboration agreement.

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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?