Reaching cruising altitude: new discovery tools to target RNA

August 31st, 2020
by Herschel Mukherjee
Scientist II, Chemistry, Arrakis
Craig Blain
Associate Director, RNA Biochemistry, Arrakis

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. In doing so, biological processes that rely on a specific target can be selectively modulated, ultimately producing the desired therapeutic effect. Understanding the […]

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

August 27th, 2020
by Jennifer Petter, PhD
Founder and CSO, Arrakis

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. We have overcome many obstacles on this mission, inventing new concepts and methods where necessary and re-engineering known concepts and methods where possible. But we have not been the only people on this […]

BioCentury This Week Podcast:
Consummating a deal under lockdown: Roche and Arrakis

May 5th, 2020
by Podcast

In a special podcast interview with BioCentury’s Editor in Chief Simone Fishburn, James Sabry, Global Head of Pharma Partnering at Roche and Mike Gilman CEO of Arrakis discuss consummating this deal while under lockdown of COVID-19.

Following our pole star to a transformative collaboration

April 8th, 2020
by Michael Gilman, PhD
CEO, Arrakis

It is an axiom of effective navigation, on earth or water or in interstellar space, that you need a fixed point to guide you. A beacon that shines clearly, never changes, never moves. If you drift off course, it pulls you back, keeps you on track. Locking your eyes on the pole star is critically […]

Arrakis achieves escape velocity

April 18th, 2019
by Michael Gilman, PhD
CEO, Arrakis

When last we spoke (here and here), Arrakis had just achieved liftoff with a $38M Series A financing in February 2017, and we were awash in fascinating and, in some cases, vexing questions. Two of my own questions that stand out upon rereading those posts from two years ago are: Is medicinal chemistry truly limited […]

Data‐driven Guideposts for Targeting RNA: Sequence, Structure and Interactions

August 1st, 2018
by Chris Burge, PhD
Professor of Biology and Biological Engineering at Massachusetts Institute of Technology

RNA’s fundamental role in biology has made it a burgeoning area for technology development and application, including as an intervention point for drug development. Virtually every step in the expression of a human gene involves RNA, from transcription through processing, localization and, ultimately, translation – the final step on the way from DNA to protein. […]

A Transition at Arrakis

June 26th, 2018
by Michael Gilman, PhD
CEO, Arrakis

Earlier this spring, Russ Petter – our Founder and CSO, very much the heart and soul of Arrakis, and a guy I’ve known for nearly twenty years – told me he had something he wanted to talk to me about and how about we do it over dinner. I didn’t think twice about it at […]

Targeting RNA – Lessons Learned from Oligos

May 8th, 2018
by James Barsoum, PhD
Senior Vice President of Biology, Arrakis

Arrakis’s mission is to drug the transcriptome.  The ability to modulate RNA biology using drug-like small molecules – we call them rSMs – will open up a vast, previously undruggable, target space.  New therapeutic capabilities will include altering the expression of proteins that cannot be targeted directly with conventional small molecules, as well as modulating […]

The Rime of the Ancient Medicinal Chemist: Structure, Structure Everywhere

April 20th, 2018
by Jennifer Petter, PhD
Founder and CSO, Arrakis

Our mission at Arrakis is to find small-molecule drugs that bind RNA, or RNA-targeted Small Molecules (rSMs). One of the articles of faith that informs and motivates this mission is that non-ribosomal RNA folds into structures that present small-molecule-compatible pockets and that those structures mediate important biological events. And, just as importantly, that binding of […]

Drug-Likeness and RNA-Targeted Small Molecules

April 1st, 2018
by Thomas Hermann
Associate Professor of Chemistry and Biochemistry at University of California, San Diego and Co-Director, UCSD Center for Drug Discovery Innovation

Only a minuscule fraction of the human genome is devoted to encoding proteins, yet proteins provide nearly 100% of the targets for currently marketed drugs. The few exceptions include antisense and RNAi oligonucleotides, some DNA-binding cancer therapeutics such as doxorubicin and cisplatin, and a chemically diverse group of antibiotics that interfere with bacterial protein biosynthesis […]

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