Codon

AUGUST 4, 2025

Protein alterations, including phosphorylation and ubiquitination, can alter a protein’s function in ways that are invisible at the level of DNA or RNA. A small molecule “barcode” is added to each droplet and attaches itself to the proteins within.

Laboratories

BioPharma Drive: Drug Pricing

AUGUST 28, 2024

Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with small molecule drugs.

RNA

BioPharma Drive: Drug Pricing

APRIL 22, 2024

billion, is the latest research collaboration involving small molecule drugs that modify RNA. The deal, which could be worth up to $1.8

RNA

Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing.

RNA

Dark Matter Blog

APRIL 4, 2023

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. Welcome to the RNA world. Familiar tools to solve familiar problems with proteins.

Small Molecule

Covalent Modifiers

MAY 10, 2023

Liganded sites were enriched for RNA-binding and protein–protein interaction (PPI) domains, including several sites found in RNP granule-forming proteins. In summary, we present a chemical strategy for the systematic discovery of condensate-modulating covalent small molecules.

Small Molecule

The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins.

RNA

Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. References Iversen et al., Front Pharmacol.,

Small Molecule

Covalent Modifiers

AUGUST 14, 2023

Our chemoproteogenomics platform integrates chemoproteomic, whole exome, and RNA-seq data, with a customized 2-stage false discovery rate (FDR) error controlled proteomic search, further enhanced with a user-friendly FragPipe interface.

Small Molecule

Dark Matter Blog

AUGUST 31, 2020

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. Our overall mission at Arrakis is to expand the set of “druggable” targets for small-molecule medicines to include RNA.

RNA

Drug Hunter

JUNE 26, 2023

This article compiles recent high-profile clinical readouts and related news with small molecules of general interest and structures where they are available. Unlike sapropterin, sepiapterin is more active in cells and is also brain-penetrant.

Small Molecule

DrugBank

MAY 22, 2024

Molecular dynamics (MD) simulations and other computational methods are vital tools in our arsenal , helping us peek into the atomic-level interactions and movements within the protein, helping reveal potential new binding sites induced by small molecule interactions. DNA and RNA are also key players, each bringing unique challenges.

Small Molecule

DrugBank

MAY 15, 2024

Researchers are experimenting with biologics—larger biological molecules that can do things small molecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells. Understanding why these targets are resistant to current therapeutic strategies is crucial.

Small Molecule

DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Multifunctional Small Molecules and Peptides Beyond PROTACs, there are other exciting tools in the new drug discovery toolbox.

Small Molecule

Dark Matter Blog

JANUARY 16, 2023

We are moving rapidly toward escape velocity in our RNA expedition at Arrakis. For those of you joining us for the first time, it’s worth a quick look back at why we decided to go all-in on the RNA world, why people thought we were crazy, and why we know we’re not. Our terra firma is small-molecule medicines.

Small Molecule

Dark Matter Blog

JUNE 21, 2023

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.

Small Molecule

LifeSciVC

JANUARY 16, 2024

Small molecule GLP1s? Small molecules are still in vogue despite IRA headwinds: We continue to hear optimism for the convenience of orals even in indications with high-commercial potential and enriched for CMS coverage (likely to fall in crosshairs of IRA).

Small Molecule

Drug Target Review

JULY 23, 2024

This laid the groundwork for my career in drug discovery using novel modalities like small molecule inhibitors, antibodies, RNA interference (RNAi) and, currently, small molecule protein degraders – which are opportunities to change how we think about medicines.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

Applying our technology has enabled us to develop a new small molecule, TT125-802, and to assign a new role to the epigenetic regulator CBP/p300 as a novel master regulator of non-oncogene resistance. This orally available small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

Small Molecule

Broad Institute

JUNE 30, 2023

The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds. That teaches us far more than looking for just a single informative data point.” Typically, studying drug therapies is an expensive and time-consuming undertaking.

Small Molecule

Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. Arrakis is the capstone of my career.

Small Molecule

Dark Matter Blog

JULY 19, 2021

However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT! This is a thing?

RNA

The Pharma Data

JANUARY 19, 2021

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

Research

Drug Target Review

DECEMBER 7, 2023

Researchers from the Laboratory of Bacteriology at The Rockefeller University have now found that bacteria sense phages by a defensive response named CBASS (cyclic oligonucleotide-based antiphage signalling system) which detects viral RNA. The novel, hairpin-shaped molecule was named cabRNA for CBASS-activating bacteriophage RNA.

RNA

Dark Matter Blog

JULY 19, 2021

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. I won’t dwell on Jim’s many accomplishments here.

Small Molecule

Sygnature Discovery

JUNE 17, 2024

Our in-house SPR equipment comprises: 2x Biacore TM 8K+ 3x Biacore TM 8K 1x Biacore TM T200 This covers mM binders, such as fragments, through to sub pM interactions (such as late stage small molecules or very high affinity biological interactions, Tollenaere et al 2023 [link] ). This technology measures affinities from mM to nM.

Small Molecule

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Pharmaceuticals

Broad Institute

JUNE 21, 2023

Cell Painting uses six fluorescent dyes to image eight different components of a cell: the nucleus, nucleoli, mitochondria, endoplasmic reticulum (ER), golgi apparatus, cytoplasmic RNA, f-actin cytoskeleton, and plasma membrane. Typically, studying drug therapies is an expensive and time-consuming undertaking.

Small Molecule

The Pharma Data

DECEMBER 21, 2020

Galidesivir treatment was associated with a more rapid decline in viral RNA levels in the respiratory tract in an apparent dose-dependent manner. About Galidesivir (BCX4430) Galidesivir, a broad-spectrum antiviral drug, is an adenosine nucleoside analog that acts to block viral RNA polymerase.

Clinical Trials

The Pharma Data

SEPTEMBER 9, 2020

Immunization of non-human primates (rhesus macaques) with BNT162b2, a nucleoside-modified messenger RNA (modRNA) candidate that expresses the SARS-CoV-2 spike glycoprotein, resulted in strong anti-viral effects against an infectious SARS-CoV-2 challenge.

Vaccine

Sygnature Discovery

JUNE 17, 2024

Diverse Target Classes We Work With: Soluble Proteins: This includes kinases, ligases, transcription factors, helicases, polymerases, and DNA/RNA modifying enzymes, among others. Membrane Proteins: Examples include cell surface receptors, GPCRs, and transporters. Nucleic Acid Targets: Focusing on specific interactions and functions.

Biophysical Assays

Codon

JUNE 11, 2024

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. Small molecules can randomly transit through the pore and create disruptions, and the polymerase ratchet mechanism is imperfect, occasionally causing DNA to temporarily reverse direction through the pore.

DNA

Drug Target Review

AUGUST 10, 2023

A twist of fate: GRP78 regulates cell migration and invasion Continuing their exploration of GRP78’s functions in the cell nucleus, the researchers utilised sophisticated RNA sequencing to compare lung cancer cells engineered to over-express nuclear GRP78 with cells lacking it.

Cell Biology

The Pharma Data

MARCH 23, 2022

Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase. But targeting Ebola virus polymerase has proven tough.

Virus

The Pharma Data

JANUARY 17, 2021

As Crooke explained, “With small molecules, if you make any chemical change, you have a new ballgame. That’s not the case with RNA-targeted drugs. “It’s the efficiency of antisense technology, coupled with its other characteristics, that makes n-Lorem’s approach feasible.”.

Disease

Elrig

APRIL 29, 2024

She has played a key role inbuilding the target identification platform and a proprietary database of transcriptome-wide, functional RNA structures. There, I supported AI-enabled DD efforts in their small molecule portfolio.

Bioinformatics

Alta Sciences

APRIL 17, 2024

Oligonucleotide-Based Techniques Most oligonucleotide therapies act through antisense mechanisms and are directed against various RNA species. Several small molecule GLP-1R agonists, such as oral orforglipron, are in late-stage clinical development.

Drug Development