Chemical Biology and Drug Design

MAY 12, 2025

This goal is achieved through an RNA-guided procedure. As the most effective gene editing method currently available, the CRISPR/Cas system has proven to be highly valuable in genomic research across a wide range of species since its discovery as a component of the adaptive immune system in bacteria.

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The Pharma Data

JUNE 25, 2025

The interim data, presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, California, offer a strong signal of therapeutic potential. a leader in RNA-targeted therapeutics. We are encouraged by the available data and eager to move salanersen into the next stage of development as quickly as possible.”

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Drug Target Review

JUNE 30, 2025

After nearly two decades at GSK, where he served as Head of Research, he left to take a bold leap into the unknown: the expanded human proteome. This allows the team to spot previously unannotated proteins and study them across multiple biological dimensions – from structure to function, from RNA expression to genetic association.

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Drug Target Review

APRIL 7, 2025

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

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The Pharma Data

JUNE 18, 2025

Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D., He holds a B.A.

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Drug Target Review

JULY 11, 2025

At the centre of this shift is 10x Genomics – a company building tools that enable researchers to study individual cells and their gene activity, both in isolation and in their original spatial context. Together, these tools give researchers a detailed view of how cells behave, where they are and how they contribute to health or disease.

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Molecular Biology Disease Therapies Bioinformatics 69

Drug Target Review

JUNE 9, 2025

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases.

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Drug Target Review

JUNE 16, 2025

1 Continuous advancements in sensitivity and spatial resolution support the role of MSI in pharmaceutical research. By integrating MSI with histopathology, clinical chemistry and DMPK data, researchers can develop mechanistic hypotheses of toxicity, improving decision making and reducing unnecessary follow-up studies. References [1].

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Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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Alta Sciences

FEBRUARY 19, 2025

Researchers must characterize the anti-drug-antibody (ADA) response in preclinical and clinical studies and report any ADA-positive samples as a risk-based approach. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

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Alta Sciences

JULY 9, 2025

What started 28 years ago as a meeting for academic researchers has evolved into a dynamic, cross-disciplinary hub for the entire cell and gene therapy ecosystem. Over just a few short days, I connected with researchers, sponsors, and partners to talk shop: industry trends, regulatory shifts, and scientific advances.

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Therapies Molecular Biology Science RNA 52

Chemical Biology and Drug Design

JUNE 9, 2025

The impact of Erianin on ox-LDL-induced injury in HUVECs was confirmed by using small interfering RNA si-Nrf-2. Matrix metalloproteinase (MMP) membrane potential was measured. Erianin pretreatment notably rescued the impaired ox-LDL-treated HUVEC viability and apoptosis and inhibited ox-LDL-induced mitochondrial dysfunction in HUVECs.

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RNA Regulations Research 100

Codon

JUNE 5, 2025

The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. This article provides a summary of the major CRISPR systems, 2 including the naturally occurring CRISPR-Cas9, -Cas12, and -Cas13 systems, as well as base editors, prime editors, and the recently uncovered bridge RNA system.

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Drug Target Review

DECEMBER 16, 2024

Today, researchers are exploring new ways to simplify and deliver CAR T cells more quickly, or even generate them directly in the patients body, an area of research led by Umoja Biopharma. As the field of cell and gene therapy continues to evolve, innovative approaches are emerging to overcome these limitations.

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The Open Targets Blog

JANUARY 30, 2025

gget consists of a collection of modules, each enabling researchers to perform common tasks in genomics, transcriptomics, and proteomics data analysis in just one line of code without exceeding the computational capabilities of a laptop.  Was there anything particularly difficult or unexpected in the process of creating gget?

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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. The statement did not specify where the non-viral delivery technology the new startup will use comes from. “We

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Broad Institute

JUNE 25, 2025

To better understand the biological processes at play in strictures, Broad Institute and Massachusetts General Hospital researchers analyzed individual cells from the intestinal tissue of Crohn’s disease patients, measuring gene activity and mapping the cells’ location in the tissue.

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addgene Blog

JUNE 17, 2025

In the research world, there is a myriad of tools this could be applied to. However, RNA interference (RNAi) remains steadfast in the background, a handy “oldie” as you might say. An oldie but a goodie — a phrase used to refer to something that has fallen out of fashion but is still useful and most importantly, effective.

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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.

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Chemical Biology and Drug Design

JUNE 9, 2025

Methylated RNA immunoprecipitation (MeRIP) and messenger RNA (mRNA) stability experiments were used to validate the influence of Wilms' tumor 1-associating protein (WTAP) on nuclear receptor coactivator-3 (NCOA3) mRNA.

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Conversations in Drug Development Trends

APRIL 15, 2025

For each condition, we highlight core biomarkers, their biological relevance, current clinical applications, and emerging opportunities for integration into research and care pathways. Mutations in genes such as SOD1, C9orf72, FUS, and TARDBP further support the role of proteinopathy and RNA dysregulation in ALS pathogenesis.

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Science Daily: Pharmacology News

JULY 2, 2024

Researchers have made a significant advance in understanding how genes are controlled in living organisms. The new study focuses on critical snippets of RNA in the tiny, transparent roundworm Caenorhabditis elegans (C. The study provides a detailed map of the 3'UTR regions of RNA in C.

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Chemical Biology and Drug Design

JUNE 25, 2025

ChIP, dual luciferase assay, and RNA pull-down were performed to explore the relation among KDM5A, miR-495-3p, and FOXO1. TTC, H&E, and TUNEL assays were applied for assessing myocardial injury and apoptosis.

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Science Daily: Pharmacology News

APRIL 5, 2024

Certain RNA molecules in the nerve cells in the brain last a life time without being renewed. RNAs are generally short-lived molecules that are constantly reconstructed to adjust to environmental conditions. Neuroscientists have now demonstrated that this.

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Science Daily: Pharmacology News

JUNE 24, 2024

Researchers have harnessed a bacterial immune defense system, known as CRISPR, to efficiently and precisely control the process of RNA splicing.

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RNA Research Disease 278

Science Daily: Pharmacology News

AUGUST 21, 2024

New research shows that rainwater could have helped create a meshy wall around protocells 3.8 billion years ago, a critical step in the transition from tiny beads of RNA to every bacterium, plant, animal, and human that ever lived.

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Science Daily: Pharmacology News

SEPTEMBER 7, 2023

By adding synergistic self-adjuvanting properties to Covid-19 RNA vaccines, researchers showed they could significantly boost the immune response generated in mice.

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Science Daily: Pharmacology News

SEPTEMBER 19, 2023

A new study shows the isolation and sequencing of more than a century-old RNA molecules from a Tasmanian tiger specimen preserved at room temperature in a museum collection. This resulted in the reconstruction of skin and skeletal muscle transcriptomes from an extinct species for the first time.

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Science Daily: Pharmacology News

NOVEMBER 21, 2024

Researchers have developed a groundbreaking microscopy method that enables detailed three-dimensional (3D) RNA analysis at cellular resolution in whole intact mouse brains.

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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

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Science Daily: Pharmacology News

JUNE 8, 2023

Researchers report that two-spot octopuses adapt to seasonal temperature shifts by producing different neural proteins under warm versus cool conditions. The octopuses achieve this by editing their RNA, the messenger molecule between DNA and proteins.

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Drugs.com

JANUARY 3, 2024

3, 2024 -- A regularly prescribed class of heart medications might be capable of treating one of the most common forms of muscular dystrophy, a new study in mice suggests.Myotonic dystrophy type 1 (DM1) is caused by abnormal RNA. WEDNESDAY, Jan.

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Science Daily: Pharmacology News

OCTOBER 9, 2024

161,979 new RNA viruses have been discovered using a machine learning tool that researchers believe will vastly improve the mapping of life on Earth and could aid in the identification of many millions more viruses yet to be characterized.

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Covalent Modifiers

FEBRUARY 25, 2024

TD19 irreversibly modifies the residues C100 and C267, preventing ALKBH5 from binding to m6A-containing RNA. Moreover, TD19 displays good anticancer efficacy in acute myeloid leukemia and glioblastoma multiforme cell lines.

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Science Daily: Pharmacology News

AUGUST 30, 2024

Researchers have uncovered a surprising genetic mechanism that influences the vibrant and complex patterns on butterfly wings. The team discovered that an RNA molecule, rather than a protein as previously thought, plays a pivotal role in determining the distribution of black pigment on butterfly wings.

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Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

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Science Daily: Pharmacology News

JANUARY 16, 2024

RNA is thought to have sparked the origin of life by self-copying. Researchers have now revealed the atomic structure of an 'RNA copy machine' through cryo-EM. This breakthrough sheds light on a primordial RNA world and fuels advancements in RNA nanotechnology and medicine.

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