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.

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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. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

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DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

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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.

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Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. 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.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

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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.

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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.

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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. With that information, researchers can map sequence changes to the spike protein to assess whether there are obvious concerns with vaccine coverage or antibody therapies.

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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: (..)

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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.

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Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. 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.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43

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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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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. CellProthera also appointed the Newcastle Centre for Life’s GMP cell therapy centre to produce the ATMP.”

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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?

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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.

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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.

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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.

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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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The Pharma Data

MARCH 23, 2022

In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. 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.

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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. Traditional companies have neither the technologies nor the financial incentives needed to develop ultra-rare therapies. We’re the middleman,” he said.

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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.

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Conversations in Drug Development Trends

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.

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The Pharma Data

MARCH 4, 2021

We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI). selective GABA A receptor agonist.

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Drug Discovery World

JUNE 6, 2023

His work has led to crucial insights into the understanding of the mechanisms by which T cells distinguish tumour cells from normal cells and has fueled the advancement of adoptive T-cell therapy approaches in various cancers, including leukemia and pancreatic cancer. From that, we’ll understand better how to use targeted therapies.”

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Drug Discovery World

FEBRUARY 29, 2024

In the first article, Reece Armstrong speaks to Natalia Mateu, Co-Founder, Jelena Aleksic, Co-Founder and Peter Curran, Chemoinformatics Scientist, about utilising 3D chemistry and AI to get small molecule therapies from the clinic to the bedside.

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Drug Discovery World

OCTOBER 17, 2023

This is an invaluable approach for precision medicine because it promises a detailed view of desired and undesired effects of treatments with cell and gene therapy agents or antibodies. The first step to reach this goal will be to generate protein and RNA data from the same sample. Let’s take CAR T cells as an example.

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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. Gene Therapy. Nucleic Acids Research.

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Codon

APRIL 30, 2023

5/ Lots O’ Gene Therapies Not one, but two applications for sickle-cell gene therapies have been submitted to the F.D.A. 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. Cell Systems.

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Codon

APRIL 30, 2023

5/ Lots O’ Gene Therapies Not one, but two applications for sickle-cell gene therapies have been submitted to the F.D.A. 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. Cell Systems.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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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.

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Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. 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. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.

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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.

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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. Typically, studying drug therapies is an expensive and time-consuming undertaking. That teaches us far more than looking for just a single informative data point.”

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