Drug Discovery World

AUGUST 16, 2023

Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. The whole world realised the power of RNA when the Covid-19 pandemic brought us the first mRNA-based vaccines.

RNA 130

RNA DNA Therapies Small Molecule 130

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 96

RNA Small Molecule Disease Assay Development 96

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 67

RNA Small Molecule Disease Research 67

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. In fact, it was these early pharmaceutical successes that gave us the confidence that we would ultimately succeed in systematically drugging a wide range of RNA structures.

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

Drug Discovery World

MARCH 26, 2024

Additional approaches, such as profiling of RNA and proteins, and the use of artificial intelligence (AI), could be key to providing a more comprehensive picture of a patient’s tumour and enable the use of optimised treatments. MT: Why was STORM attending the conference? We were there to present the latest findings from our research.

Therapies 163

Therapies Small Molecule RNA DNA 163

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 52

Small Molecule FDA Approval FDA Drugs 52

Drug Discovery World

JULY 20, 2023

Arrayjet and Chemspace have agreed a partnership to augment and streamline Chemspace’s small molecule microarray (SMM) service offering. The technique is suited for targeting proteins and structured RNA, developing heterobifunctional degrader molecules (e.g., PROTAC), and training AI drug discovery models.

Small Molecule 100

Small Molecule Assay Development Compound Screening RNA 100

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 52

RNA Small Molecule DNA Licensing 52

Drug Discovery World

AUGUST 29, 2024

For example, multiple RNA helicases are being targeted for cancer and viral infections before their catalytic properties are well understood. Fortunately, their unwinding activity is coupled to ATP hydrolysis, and homogenous ADP detection methods developed for kinase screening are being adapted to screen and profile helicase inhibitors.

Small Molecule 148

Small Molecule Drugs Protein Expression Assay Development 148

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible. .

Small Molecule 246

Small Molecule Pharmacy RNA Packaging 246

Drug Discovery World

MAY 26, 2023

The top stories: Biopharma investment drops by nearly 50% in 2022 Cell therapy, RNA technology, antibody drug conjugates and AI/machine learning are among the key areas of investment for pharma, according to a new report. million to develop the next generation of small molecule drugs against disease areas of high clinical need.

Small Molecule 130

Small Molecule DNA Government RNA 130

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 52

Small Molecule RNA Drugs International 52

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 84

Small Molecule Clinical Trials Drugs Therapies 84

Drug Discovery World

JANUARY 13, 2023

Alliance to develop circular RNA-based therapeutics . Esperovax and Ginkgo Bioworks have agreed a partnership to develop circular RNAs (circRNAs) for a variety of therapeutic applications. . The top stories: . Collaboration to identify up to 10 novel target-antibody pairs . Cambrian expands fibrosis pipeline .

Small Molecule 130

Small Molecule RNA Drugs Engineering 130

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 67

Small Molecule RNA DNA Pharmaceutical Companies 67

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 130

Small Molecule RNA Trials Treatment 130

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 83

Small Molecule Molecular Biology Drugs Disease 83

Drug Discovery World

FEBRUARY 14, 2024

However, microRNAs do not code for proteins, so it wasn’t clear how such small molecules can make changes to the skin. “We Dr Svitlana Kurinna, University of Manchester Division of Cell Matrix Biology and Regenerative Medicine and lead investigator of the study, said: “We had data showing microRNAs can regulate skin growth.

Small Molecule 130

Small Molecule Regulations RNA Drugs 130

Drug Discovery World

MAY 13, 2024

Key to this is the ability to rapidly determine protein structures alone and in combination with antibodies or small molecules. They can also look at impact of sequence changes to proteins like Mpro to assess utility or future design iterations for anti-viral small molecule.

Vaccine 148

Vaccine Virus Small Molecule Disease 148

Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

RNA 148

RNA Small Molecule Vaccine FDA Approval 148

Drug Discovery World

DECEMBER 26, 2023

Otsuka Pharmaceutical and Ionis Pharmaceuticals Otsuka Pharmaceutical and RNA therapy company Ionis have entered into a licence agreement for Otsuka to acquire exclusive marketing rights to Ionis’ hereditary angioedema (HAE) drug candidate donidalorsen in Europe.

Small Molecule 130

Small Molecule Drugs Molecular Biology Bioinformatics 130

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 52

Small Molecule RNA Fragment Screening DNA 52

Drug Discovery World

DECEMBER 21, 2023

Three trends in the antibody-drug conjugate (ADC) market Antibody-drug conjugates are biopharmaceutical products in which a monoclonal antibody (mAB) is linked to a small molecule drug with a stable linker. Read the article: Where are the hottest drug discovery hubs in the US?

FDA Approval 148

FDA Approval Therapies Small Molecule Clinical Trials 148

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 52

Small Molecule RNA 52

Drug Discovery World

APRIL 6, 2023

from Fabien Vincent, Pfizer, Groton, US High content phenotypic and pathway profiling identifies novel drug mechanisms-of-action tailored towards cancers of unmet need by Neil Carragher, University of Edinburgh, Edinburgh, UK Functional genomics as an enabling tool for phenotypic screening by Matthew Garnett Chemistry to the Clinic: Part 1-3: (..)

Small Molecule 130

Small Molecule Targeted Protein Degradation Clinical Trials Engineering 130

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 59

RNA Immune Response DNA Molecular Biology 59

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 52

Small Molecule RNA DNA Therapies 52

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 52

Small Molecule RNA Clinical Trials DNA 52

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 140

Small Molecule Trials Therapies Disease 140

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 52

RNA Small Molecule Science Therapies 52

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 52

Small Molecule RNA Science Therapies 52

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 52

Biophysical Assays Screening Hits Fragment Screening Assay Development 52

Drug Discovery World

APRIL 25, 2023

The new life science companies BII has accepted into theVentureLabacceleration program are: AIDA Oncology which works to select the best cancer drug for each patient, using the RNA expression pattern of their tumour and machine learning.

Science 130

Science Small Molecule Disease Government 130

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 52

RNA Small Molecule Synthetic Chemistry Science 52

The Pharma Data

MARCH 7, 2022

In their work, Levy and her colleagues focused on a complex of proteins called PRC2 that silences genes by attaching a small molecule, called a methyl group, to a protein that packages genes called histones. Cas9 binds and uses RNA as an address-tag. The AI-designed blocking protein was the cargo of the dCas9-RNA construct.

RNA 52

RNA DNA Packaging Small Molecule 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). Today, there are several other categories of RNA therapeutics with drugs already approved or currently in development.

Therapies 130

Therapies RNA Vaccine Small Molecule 130

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases.

Drugs 190

Drugs Science Therapies Targeted Protein Degradation 190