As a small biotech company with new technology at the frontier of the known drug discovery universe, it may feel like you’re charting a course alone in a dark, cold, and empty void with hailing frequencies open, but with no incoming transmissions. But you would be wrong. If you’re intentionally equipped to locate, identify, and […]
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. This typically meant engaging with their functional pockets and thereby blocking their function. The concepts […]
We are moving rapidly toward escape velocity in our RNA expedition at Arrakis. We want to mark this progress with some updates and perspectives in the Dark Matter blog. In the months ahead, we are rolling out a series of posts called “Fearlessly Forward” to bring you up to speed on how we’ve moved forward […]
Arrakis has gone through many significant transitions since its founding in 2015. We’ve evolved and matured scientifically. We’ve added investors and partners. And, of course, people come and go. Today, we reach a bittersweet transition at Arrakis. We bid our head of research and employee #005, Jim Barsoum, au revoir, as he marches bravely into […]
Back in 2015, I felt compelled to set out in a new direction in drug discovery. I was in my third year as VP of Chemistry for Celgene. This was a great gig – good people, good science. However, it required a level of travel that began to seem excessive. My kids were old enough […]
49 years ago, when I was 16 years old, I wanted to do exactly what I am doing now. The only subject in school that held my interest was biology. As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. I wanted to use molecular biology to create drugs. […]
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 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 […]
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.
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 […]
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 […]
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. […]
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 […]
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 […]
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 […]
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 […]
Though not nearly as famous as its double-stranded cousin, RNA is a fascinating molecule and its potential as a drug discovery substrate continues to unfold beyond merely transferring information from DNA into proteins. To perform its role in the cell, RNA must move, contort and adapt to a wide range of functional and regulatory requirements. […]
Small molecules that bind to macromolecular nucleic acids are well known. Before the 1960s, dyes such as aminoacridines, were utilized by histologists and biologists to image specific sub-nuclear structures.1 This was perhaps the earliest recognition that small molecules can bind macromolecular nucleic acids. In 1961, the first nucleic acid-small molecule binding hypothesis (the “intercalator hypothesis”) […]
In “The Right Stuff,” Tom Wolfe noted that astronauts were granted hero status before their missions, perhaps because no one expected them to return. Happily, Arrakis also has received a lot of laudatory attention just for launching the mission of drugging RNAs with small molecules – before we’ve even achieved it. The plaudits are lovely, […]
Perspectives from the ‘Targeting RNA Using Small Molecules’ meeting @NYASciences #rSMs Our team at Arrakis Therapeutics recently had the pleasure to co-sponsor the ‘Targeting RNA Using Small Molecules’ symposium at the New York Academy of Sciences. While several conference sessions over the years have focused on our favorite topic, including the (in)famous Gordon Conference that […]
As we battle to expand the reach of modern therapeutics to treat diseases with high unmet need, we confront the limitations in our armamentarium. Most often we are limited by biology – we don’t understand the molecular drivers of disease and consequently don’t know which targets to aim at. But the more frustrating situation is […]
So here we are. Nearly two years in the making, much of it focused, fastidious work by founder Jennifer Petter (of whom much more below), capped by an exhilarating and exhausting sprint to the finish by an outstanding team, Arrakis Therapeutics has now lifted off. Today we announced a $38M Series A financing led by […]
Most old-timers in drug discovery will agree on one thing: It never gets easier. Sure, maybe we learn a little from our mistakes along the way and the toolkit gets steadily flashier. But the easy targets, if ever they were truly easy ones, are long gone. We are left with either inscrutable biology or, even […]
