Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

RNA

Drug Target Review

JUNE 17, 2025

The dynamic behaviour of RNA transcripts, the regulation and modification of proteins, the lipid composition of membranes, the structural organisation of cells and the spatial context of tissues all play critical roles in disease progression and therapeutic response. There are already early examples of this potential in action.

RNA

Broad Institute

MARCH 12, 2025

Their method, known as Perturb-FISH, combines imaging-based spatial transcriptomic measurements with large-scale detection of CRISPR guide RNAs. In addition, the research team tested the method in a more complex system, using a xenograft model developed in the Broad Cancer Program. Paper cited Binan L, et al.

RNA

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.

Treatment

Drug Target Review

JULY 24, 2025

From academia to antibody architect JJ’s scientific path began in neurobiology with a PhD followed by a postdoctoral role at the Genome Research Center at Academia Sinica. That shift into translational research changed the direction of his career. He earned his Ph.D. in Molecular Medicine from National Taiwan University.

Immune Response

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.

Disease

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.

Clinical Development

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.”

Therapies

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.

Molecular Biology

Codon

JULY 28, 2025

Researchers have identified nearly 10,000 monogenic diseases , or conditions caused by errors in a single gene. Researchers cannot extract, edit, and replace neurons the way they do blood cells, so diseases involving the brain, muscles, or liver require vectors that reach and fix cells in their natural environments.

Immune Response

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.

RNA

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.

Disease

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].

Drugs

Drug Target Review

JULY 21, 2025

In Part 2 of our conversation with Layla Hosseini-Gerami, Chief Data Science Officer at Ignota Labs , we explore the many forms of toxicity and how AI-powered tools like omics and cell painting are transforming early prediction in pre-clinical research. How can we understand what the consequences of that are?”

Drugs

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).

RNA

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.

Drug Development

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.

Therapies

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.

RNA

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.

DNA

Codon

NOVEMBER 3, 2024

Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB. Chilean research participants aged 15 to 35 who received the vaccine during a case-control study , for example, only showed a 10 percent reduced risk of pulmonary TB.

Vaccine

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.

Therapies

Broad Institute

FEBRUARY 26, 2025

Why brain cancer is often resistant to immunotherapy By Allessandra DiCorato February 26, 2025 Breadcrumb Home Why brain cancer is often resistant to immunotherapy Researchers find four coordinated gene expression programs in immune cells from glioma tumors, including two that could lead to immunotherapy resistance.

Immune Response

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?

Bioinformatics

Codon

APRIL 14, 2025

While I deeply respect that tolerance, it can keep the field stuck: if we see pain as par for the course, it’s easy to overlook chances to make biological research faster, cheaper, and more efficient. Of course, biological research is shaped by different pressures than hardware projects.

Protein Expression

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

RNA

Codon

NOVEMBER 1, 2024

Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. Still, many of the bottlenecks slowing biology today are biophysical , rather than computational.

DNA

BioPharma Drive: Drug Pricing

JULY 16, 2025

In a statement Monday , Sarepta said it plans to cut approximately 36% of its staff and pause research on several drug programs. Elevidys is the product of many years of research into the deadly neuromuscular condition and, in 2023 , became the first and currently only gene therapy for the disease approved in the U.S.

Therapies

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.

Disease

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.

RNA

BMG Labtech

DECEMBER 20, 2024

Absorbance measurements, often referred to as light absorbance, are so versatile and widespread that they are one of the first techniques many life science researchers encounter in a practical setting. Nucleic acid quantification Researchers are often faced with the need to quantify nucleic acids in samples.

DNA

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

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.

RNA

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.

RNA

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.

Clinical Trials

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.

RNA

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.

RNA

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.

RNA