Drug Target Review

JANUARY 2, 2024

“We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun. Scrutinising these distinctions aids scientists in comprehending cellular operations, thereby fostering enhanced therapeutic approaches for diverse diseases.

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Drug Target Review

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What makes the potential therapeutic for Huntington’s disease unique compared to other treatments?

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Drug Target Review

DECEMBER 6, 2023

Dear readers, RNA, or ribonucleic acid, plays a pivotal role in the intricate dance of cellular processes. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.

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RNA DNA Vaccine Drug Development 52

ASPET

JULY 19, 2023

Ras- G TPase-activating protein (SH 3 domain)- b inding p roteins (G3BP) are RNA binding proteins that plays a critical role in stress granule (SG) formation. Recent evidence suggests that G3BPs can also regulate mRNA expression through interactions with RNA outside of SGs.

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RNA Virus Regulations Disease 100

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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Treatment Clinical Trials Small Molecule Therapies 81

Drug Target Review

JULY 5, 2023

In nature, proteins take millions and trillions of years to evolve, 1 but scientists do not have this time when designing drugs for life-threatening diseases. Therefore, researchers use directed protein evolution technologies , to evolve and design proteins with specific functions under well-defined conditions and a practical time frame.

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RNA Engineering Disease Drugs 97

Drug Target Review

JULY 24, 2023

Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The study suggests that this regulatory system could be a potential target for future malaria treatments, offering hope for combatting this deadly disease that affects millions worldwide.

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RNA Regulations Bioinformatics Laboratories 98

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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Small Molecule RNA Clinical Trials Drugs 52

Codon

AUGUST 26, 2024

Credit: OpenStax College The innate immune system has been evolutionarily successful for three reasons: its tools target the most fundamental properties of viruses , act as prophylactics —meaning they prevent diseases, rather than treat them—and are widespread. Existing medicines only pose a partial solution.

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RNA Virus DNA Vaccine 97

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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The Pharma Data

FEBRUARY 8, 2021

The amended protocol being implemented for all ongoing adult and adolescent fitusiran clinical studies was presented today at the 14 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

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RNA Treatment Vaccine Therapies 52

Chemical Biology and Drug Design

SEPTEMBER 15, 2023

Abstract Chronic rhinosinusitis (CRS) is an inflammatory disease of paranasal sinuses. CRS rabbit models were constructed by Staphylococcus aureus infection and rabbits were injected with lentiviral vectors of short hairpin RNA-targeting Chemerin (shChemerin), followed by micro-computed tomography (CT) scan.

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RNA Treatment Disease Research 100

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. tuberculosis.

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DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. However, mRNA technology is not limited to infectious diseases. Once delivered into the body, the mRNA instructs cells to produce these antigens, which are then presented to the immune system.

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Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

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RNA Regulations Disease DNA 98

DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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KIF1A

SEPTEMBER 16, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This is also important when newborns present as having a “common” disorder that may have a rare genetic component, like KIF1A in genetic cerebral palsy.

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Regulations RNA Disease Research 105

The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

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Treatment Therapies DNA Trials 52

Broad Institute

OCTOBER 28, 2024

So we took lots of reference single-cell RNA sequencing data from atlases and used our scalable machine learning algorithms to embed all of the gene expression data on these cells into compact vector representations — you can think of these as a signature for each cell. We wanted to build a tool like that for cell biology.

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Engineering Cell Biology Disease RNA 119

Broad Institute

AUGUST 19, 2024

Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Jude’s, the team identified a group of genes involved in processing uLCFAs that are present only in the non- Lactobacillus iners species. At the Ragon Institute, Kwon’s lab is working to move this research toward a human clinical trial. “We

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The Pharma Data

JULY 12, 2021

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the presentation of data from the company’s HIV clinical development program at the 11th International AIDS Society Conference on HIV Science (IAS 2021) from July 18-21. Late Breaking Oral Presentation OALC01LB03. On-Demand Oral Presentation OAB0304.

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Pharmacokinetics Clinical Trials Trials Treatment 52

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

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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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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. Please summarise Eterna Therapeutics’ ($ERNA) new research at ASGCT on a cell therapy approach that integrates multiple cell types to emulate the natural immune system’s fight against diseases?

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Therapies DNA Engineering Virus 103

KIF1A

SEPTEMBER 9, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. To investigate these proteins, the authors used RNA interference to silence Nup153 and KIF1A.

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RNA Treatment Research Disease 105

Codon

DECEMBER 12, 2024

It’s important to note that the 300-page report, while presenting detailed evidence for the potential harms of mirrored life, uses painstakingly speculative language. DNA and RNA molecules are also built from exclusively right-handed nucleic acids. It gives humanity time to steer away. This is one of them.

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RNA DNA Vaccine Virus 102

Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration. However, the mechanism of this observation was not fully understood.

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RNA Cell Biology Clinical Development Disease 64

The Pharma Data

MARCH 8, 2021

The data were presented at the 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). In patients with chronic kidney disease, assess serum phosphorus. In patients with chronic kidney disease, assess serum phosphorus. Pregnancy and lactation. Source link:[link].

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Treatment Therapies RNA Laboratories 52

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases. This can potentially correct the root causes of some diseases at the genetic level.

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The Pharma Data

MARCH 8, 2021

These preliminary findings were presented today during Science Spotlights TM at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021). Findings from the primary efficacy and safety endpoints and additional secondary objectives will be presented at an upcoming medical meeting.

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Trials Virus RNA Research Laboratories 69

Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. However, they also limit beneficial responses by suppressing sterilising immunity and limiting antitumour immunity.

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Government DNA RNA Regulations 104

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. The German study is also scheduled for presentation on Saturday at the American Society of Hematology’s virtual annual meeting.

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Therapies Disease Medical Schools Virus 52

Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections.

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Hospitals Virus Medical Schools Science 137

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape.

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The Pharma Data

NOVEMBER 10, 2021

By creating genetically finagled mouse models and doing single cell RNA-sequencing, Feng and associates plant that Shp2 was needed by excrescence cells for HCC development, which validated and corroborated allowing that Shp2 presented an ideal target for new cancer curatives. ” Source link: [link].

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Disease Molecular Biology RNA Drugs 52

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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DNA Laboratories Hospitals Virus 97

Drug Target Review

SEPTEMBER 25, 2024

5 Here, the original oncogene is still present but the transdifferentiated cancer cells are less dependent on it. Investigating non-oncogene resistance as a parallel defense mechanism used by cancer cells to evade therapies could therefore present a significant opportunity to address drug resistance more comprehensively.

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