Codon

JULY 28, 2025

Researchers have identified nearly 10,000 monogenic diseases , or conditions caused by errors in a single gene. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures.

Immune Response

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.

Vaccine

BioPharma Drive: Drug Pricing

JUNE 24, 2025

Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. Earlier this year, it laid off 15% of its workforce.

RNA

Broad Institute

JUNE 25, 2025

Related news Single-cell analysis of Crohn’s disease reveals a detailed picture of inflammation in the gut Up to half of patients with Crohn’s disease, an inflammatory bowel disease, develop a complication called fibrosis, where the gut becomes scarred and obstructed, causing pain and bloating.

Disease

Twist Bioscience

APRIL 3, 2025

Human Comprehensive Exome Human Core Exome Human Methylome Panel Human RefSeq Panel Mitochondrial Panel Mouse Exome Panel Respiratory Virus Research Panel Comprehensive Viral Research Panel Twist's Whole Exome Library Preparation Workflow.

RNA

New Drug Approvals

JULY 18, 2025

3] It was discovered for the treatment of hepatitis C [4] and has since been investigated for use against other viral diseases such as AIDS and COVID-19 , [2] [5] for which it was granted conditional approval in China. [6] 11] It is believed to work by inhibiting the RNA-dependent RNA polymerase (RdRp) enzyme in the SARS-CoV-2 virus. [12]

RNA

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. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

RNA

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.

RNA

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.

RNA

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.

RNA

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.

RNA

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.

Regulations

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.

Vaccine

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.

Treatment

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

Hospitals

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.

Engineering

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.

Pharmacokinetics

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.

Small Molecule

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.

Small Molecule

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?

Therapies

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.

RNA

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].

Treatment

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.

Trials

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.

Government

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.

RNA

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.

Small Molecule

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.

FDA Approval

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.

Small Molecule

The Pharma Data

JULY 12, 2021

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced today the presentation of previously announced Phase 2 interim results from two Phase 2/3 clinical trials (MOVe-OUT and MOVe-IN) of molnupiravir (MK-4482/EIDD-2801), an investigational oral antiviral therapeutic. About Molnupiravir.

Clinical Trials

Broad Institute

SEPTEMBER 26, 2024

Morphological profiling of cells is commonly employed in drug discovery across many disease areas. However, there has been limited study on its application specifically in the development of therapeutics for heart diseases. The Broad Institute almost doesn’t feel real to me, it is special and unlike any place I've ever visited.

RNA

New Drug Approvals

SEPTEMBER 6, 2024

US10071079, Example 15 Ref WO2018005586 [link] The invention further pertains to pharmaceutical compositions containing at least one compound according to the invention that are useful for the treatment of conditions related to TLR modulation, such as inflammatory and autoimmune diseases, and methods of inhibiting the activity of TLRs in a mammal.

RNA

The Pharma Data

OCTOBER 14, 2020

Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Pharmaceuticals

The Pharma Data

JULY 6, 2021

New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. Oral presentation. These data support the revised fitusiran dose and dosing regimen implemented in the ongoing adult and adolescent studies.

Therapies

DrugBank

JANUARY 29, 2025

Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis. In sickle cell disease, for instance, a missense mutation in the HBB gene , encoding the beta-globin subunit of hemoglobin, leads to the production of abnormal hemoglobin S.

Therapies

PerkinElmer

JANUARY 19, 2020

Here are ten key tutorials, presentations and posters designed to help you achieve fast, accurate and reproducible results in precision medicine, next generation sequencing, homogenous assays and more. Time away from the lab is limited and valuable. Here we describe a cloud-enabled AI platform for analyzing and visualizing HCS data.

International

Drug Target Review

SEPTEMBER 28, 2023

They needed reference materials for the disease in order to develop and validate diagnostic tests. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. NGS tools can be used to ‘read’ DNA to identify a disease subclass and detect specific mutations.

DNA

The Pharma Data

MARCH 28, 2022

We are incredibly grateful for the selfless commitment of the individuals with ALS who participated in the study, and the community’s dedication to advancing research for this devastating disease,” said Toby Ferguson, M.D., The companies will present the BIIB078 Phase 1 data at a future scientific forum. Biogen Inc.

RNA