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

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

DrugBank

MAY 22, 2024

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

Small Molecule

DrugBank

MAY 15, 2024

This method was more about serendipity than science. Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. But as molecular biology has advanced, so too has our approach to finding new drugs.

Small Molecule

Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Pushing the boundaries of science always opens the door to new, impactful developments, although there are always challenges along the way.

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

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

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

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

Dark Matter Blog

JULY 19, 2021

This was a great gig – good people, good science. 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. Raj Parekh, a partner at Advent Life Sciences, had been thinking about this problem for a year or so.

RNA

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

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. Don’t be a stranger!

Small Molecule

Sygnature Discovery

JUNE 17, 2024

Our skilled protein science team can provide in-house production of proteins to support all these assay formats if needed. Microscale Thermophoresis (MST) Microscale Thermophoresis (MST) is a technology that uses fluorescently labelled proteins to analyse small molecule binding in solution.

Small Molecule

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. Rising from Research Scientist to Senior Scientist, I supported early-stage DD efforts within their Protein Science group, based in Cambridge, UK.

Bioinformatics

Drug Target Review

AUGUST 10, 2023

Published in the Proceedings of the National Academy of Sciences , a new discovery from the University of Southern California (USC) on cancer metastasis has opened up new possibilities for combating the spread of this devastating disease.

Cell Biology

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. Now equipped with more serviceable proteins and clever methods to slow down DNA traffic, nanopores could finally evolve from a niche science project to an industrial sequencing workhorse.

DNA

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. billion doses by the end of 2021.

Vaccine

The Pharma Data

JANUARY 17, 2021

“I’m still involved with the science that we created and continue to advance,” he told BioSpace. 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.

Disease

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development

KIF1A

AUGUST 20, 2024

Susannah is being treated with an Antisense Oligonucleotide (ASO) : To break down the term, this is a molecule designed with a sequence of 20 (oligo, “few”) letters of DNA (nucleotide) that match a sequence on the mutant KIF1A RNA (Antisense) and knock it down. With gratitude, in the Year of Pioneers.

Science

Dark Matter Blog

APRIL 8, 2020

Most people, and in particular most investors we spoke to in those early days, thought we were nuts – that RNA lacked the structural and molecular complexity that medicinal chemistry exploits with such great effect for proteins. One is that molecular recognition is purely a matter of physics and RNA has to play by the same rules.

RNA

The Pharma Data

OCTOBER 21, 2020

AT-527 is expected to be ideally suited to combat COVID-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”.

RNA

Codon

APRIL 2, 2023

Basic Science *No evidence for a common blood microbiome based on a population study of 9,770 healthy humans. Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). Massively parallel profiling of RNA-targeting CRISPR-Cas13d.

DNA

Codon

MARCH 20, 2023

Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Marine biofilm engineered to produce current in response to small molecules. Science Advances. Science Advances. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.

Virus

Broad Institute

SEPTEMBER 23, 2024

Using what they learned from these experiments, they developed a framework — ligation-enabled messenger RNA-oligonucleotide assembly, or LEGO — that enables researchers to chemically modify the structure of mRNA molecules and influence their interactions with the cell's protein-translation machinery, achieving desired therapeutic effects.

RNA

The Open Targets Blog

APRIL 5, 2023

The Druggable Genome The Druggable Genome is the subset of all human genes encoding proteins that could be targeted with a small molecule or antibody drug [4]. We then designed our own CRISPR guide RNA library against the druggable genome and transduced the corresponding lentiviruses into millions of our iNeurons.

Small Molecule

Codon

APRIL 30, 2023

Read more at Science. Science abstracts were pulled from five different medical journals and then fed into ChatGPT. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. Science Advances. Ann Gibbons for Science. Zhou et al. Cell Systems.

Vaccine

Codon

APRIL 30, 2023

Read more at Science. Science abstracts were pulled from five different medical journals and then fed into ChatGPT. Read Ribosomes are able to build proteins because they contain a strand of RNA that imbues them with their catalytic power. Science Advances. Ann Gibbons for Science. Zhou et al. Cell Systems.

Vaccine

Codon

DECEMBER 12, 2024

” The report accompanies an article in Science , also released today, entitled “Confronting Risks of Mirror Life.” For the article in Science , more than 30 scientists from ten countries are calling on the broader community to confront the risks of mirrored life. ” But what, exactly, is a mirrored organism?

RNA

Codon

APRIL 16, 2023

Read Basic Science *Noncoding translation mitigation. Science Advances. Read Open science and data sharing in cognitive neuroscience with MouseBytes and MouseBytes+. Read An RNA-based system to study hepatitis B virus replication and evaluate antivirals. Science Advances. Science Translational Medicine.

Engineering

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Science Advances.

DNA

Codon

MAY 30, 2023

The researchers first compared the editing efficiency of different versions of IscB when coupled with 'ωRNA,' which guides the enzyme to the right spot on the DNA. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome. Science Advances.

DNA

The Pharma Data

SEPTEMBER 8, 2020

LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. About AstraZeneca.

Science

The Pharma Data

SEPTEMBER 8, 2020

About AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

Science

Codon

MARCH 26, 2023

Link Basic Science *Genomic analyses of hair from Ludwig van Beethoven. Link Experimental Tests of the Virtual Circular Genome Model for Nonenzymatic RNA Replication. Link Single-molecule visualization of stalled replication-fork rescue by the Escherichia coli Rep helicase. Science Advances. Science Advances.

DNA

Codon

APRIL 8, 2025

Experiments performed with the bacterium Streptococcus pneumoniae not only led to the discovery of DNA as the principal hereditary molecule but also yielded early tools for genetic engineering. The study of RNA tumor viruses revealed reverse transcriptase , an enzyme integral in studying RNA biology.

RNA

Codon

MAY 29, 2025

While small-molecule drugs continue to play a critical role in modern medicine, we are witnessing a shift toward the increasing development of amino acid-based therapeutics. This deadly structure allows it to kill its victims by preventing their cells from making messenger RNA (mRNA) through inhibition of the enzyme RNA Polymerase II.

Drugs