DrugBank

MAY 15, 2024

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. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.

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Small Molecule Molecular Biology Drugs Disease 82

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.

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Small Molecule RNA Drugs Disease 52

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.

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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.

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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.

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Drug Target Review

JULY 23, 2024

Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. TPD engages the body’s natural protein recycling system by selectively eliminating disease-causing proteins, ultimately addressing the root cause of disease.

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Small Molecule Targeted Protein Degradation Science Disease 115

Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

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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.

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Small Molecule Therapies Regulations Treatment 105

New Drug Approvals

SEPTEMBER 29, 2024

link] 01 Aug 2022 Cortexyme is now called Quince Therapeutics You need to be a logged in or subscribed to view this content This small molecule is an orally available protease inhibitor targeting the lysine proteases of the periodontal pathogen Porphyromonas gingivalis. gingivalis is associated with cardiovascular disease.

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LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Small molecule GLP1s? Kidney disease: outcomes in CKD are likely to benefit from weight loss as well, with the semaglutide Phase 3 FLOW trial stopped early last October after an interim efficacy read (full data expected this year).

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Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

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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.

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Broad Institute

JUNE 21, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. The researchers also looked at the tool’s ability to detect changes in cell morphology after exposure to drugs and small molecule compounds.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We they can help many patients.”.

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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.”.

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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. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. Szymczak P. Nature Communications.

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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.

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The Open Targets Blog

APRIL 5, 2023

I asked him to tell me more about the project and its implications for target identification and prioritisation in neurodegenerative diseases. One treatment angle for neurodegenerative diseases is to dampen the Unfolded Protein Response to rescue the neurons. How did you get the idea for this project?

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The Pharma Data

MARCH 7, 2022

The approach will allow researchers to understand the role individual genes play in normal cell growth and development, in aging, and in such diseases as cancer, said Shiri Levy, a postdoctoral fellow in UW Institute for Stem Cell and Regenerative Medicine (ISCRM) and the lead author of the paper. Cas9 binds and uses RNA as an address-tag.

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Codon

APRIL 2, 2023

Read A new DNA polymerase variant, called RT-KTq I614Y, can directly detect RNA modifications, including pseudouridine (Ψ) and queuosine (Q). By combining this new variant with standard sequencing methods, it’s possible to identify RNA modifications in a really simple way. Nucleic Acids Research. Communications Biology.

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The Pharma Data

JUNE 26, 2021

“Our vision is to harness the power of the immune system against cancer and infectious diseases. They feature the Company’s proprietary RNA-lipoplex delivery formulation which is designed to enhance stability and translation of the mRNA cargo as well as specifically target dendritic cells. About BioNTech.

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Codon

AUGUST 27, 2024

Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. Sometimes, though, novel diseases do emerge, and as COVID-19 demonstrated, they can surprise us. This is the third essay of four in our pandemic mini-issue.

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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.

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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.

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Alta Sciences

APRIL 17, 2024

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. We have conducted over 130 in-life studies in the last five years (over 95% conducted in NHPs), including IND-enabling studies for rare diseases and CRISPR gene editing therapeutics in NHPs.

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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.

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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. Read more in Nature Methods.

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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. Read more in Nature Methods.

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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. The major challenge in developing drugs directly against Ebola virus polymerase is that scientists have not solved its atomic structure.

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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. Argentina and Brazil.

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Codon

APRIL 30, 2023

If these printers were set up in resource-starved countries, where malaria and other diseases are endemic, they could make a world of difference. If approved, these wouldn’t be the first gene therapies on the market, but they would be the first gene therapies for a disease that affects something like 100,000 Americans.

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Codon

APRIL 30, 2023

If these printers were set up in resource-starved countries, where malaria and other diseases are endemic, they could make a world of difference. If approved, these wouldn’t be the first gene therapies on the market, but they would be the first gene therapies for a disease that affects something like 100,000 Americans.

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Codon

JUNE 11, 2024

But these little machines, small enough to fit inside a pocket, have become commonplace. Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. Such a device seems implausible.

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The Pharma Data

MARCH 4, 2021

Our work together demonstrates the strength of Takeda’s partnership model and our commitment to delivering transformative medicines to patients with neurological diseases.”. We will advance and enrich our pipeline while continuing to build a leading company in rare diseases of the brain. President of Research and Development at Takeda.

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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.

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Conversations in Drug Development Trends

MAY 30, 2024

The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.

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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. These results suggest that early antiviral treatment of SARS-CoV-2 infection may protect against developing severe COVID-19 lung disease. government agencies and other institutions.

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