Broad Institute

OCTOBER 30, 2024

Researchers have typically analyzed these cells as a mixed-together group, but this approach can miss rare cell types, and makes it difficult to draw conclusions about how cells interact to drive the disease. They also applied four spatial transcriptomics methods, including Slide-Seq and MERFISH, to a subset of the biopsies.

Research

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

Clinical Development

Broad Institute

MARCH 12, 2025

By Leah Eisenstadt March 12, 2025 Credit: Broad Communications Scientists in the Spatial Technology Platform at the Broad develop, use, and share spatial -omics approaches, such as Perturb-FISH, that can reveal the roles of genes in various cell types and the impact of intercellular interactions on health and disease.

RNA

Drug Target Review

JUNE 30, 2025

“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. Today’s broad-spectrum drugs often miss the mark, especially for chronic diseases like COPD or heart failure.

Disease

The Pharma Data

JUNE 25, 2025

The goal is to boost SMN protein production more effectively and offer a more convenient, once-a-year dosing schedule , thereby improving long-term disease management and patient compliance. These reductions were sustained through one year , highlighting salanersen’s potential to meaningfully slow disease progression. Source link

Therapies

Drug Target Review

JULY 11, 2025

Neurological diseases are among the most complex and least understood conditions in medicine. Despite the knowledge that certain genes increase the risk of contracting diseases like Alzheimer’s and Parkinson’s, it is still not fully understood how those diseases develop in the brain. But that is beginning to change.

Molecular Biology

The Pharma Data

JUNE 6, 2025

These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. These designations underscore rilzabrutinib’s potential to offer a meaningful therapeutic advance for patients with limited treatment options.

Disease

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.,

RNA

The Pharma Data

AUGUST 1, 2025

The agreement centers around the investigational RNA interference (RNAi) therapy, plozasiran, which targets apolipoprotein C-III (APOC3) and is under development for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). President and CEO of Arrowhead. “We

Licensing

LifeSciVC

JUNE 12, 2025

Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing.

RNA

Drug Target Review

JUNE 16, 2025

Multiomics MSI studies provide crucial insights into drug pharmacology and toxicity, supporting the development of innovative therapies including chimeric antigen receptor (CAR) T-cell treatments, oligonucleotide therapeutics, antibody drug conjugates (ADCs) and ribonucleic acid (RNA) therapeutics. Bioanalysis 2019, 11 (11), 1099–1116.

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

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

The Pharma Data

JUNE 9, 2025

Alnylam Secures European Commission Approval for AMVUTTRA® to Treat Cardiomyopathy in ATTR Amyloidosis Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has received European Commission (EC) approval for an expanded indication of its RNAi drug AMVUTTRA® (vutrisiran).

Treatment

Codon

JUNE 5, 2025

Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.” ” Despite its association with genome editing, 1 CRISPR didn’t start out as a tool for fighting genetic disease.

DNA

Drug Target Review

DECEMBER 16, 2024

For patients with aggressive diseases, the waiting time for CAR T-cell treatment may be too long. Lentiviral platforms offer the potential of a one-time treatment to generate long-lasting CAR T cells in a patients body, while other RNA-based platforms offer an approach that uses repeat dosing for short-term generation of CAR T cells.

Therapies

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. Can We Lay This Scourge to Rest?

Vaccine

The Open Targets Blog

JANUARY 30, 2025

Amongst other tasks, gget opentargets can quickly find diseases and drugs associated with a specific gene. Joe is a fourth-year USC-Caltech MD-PhD student in the Pachter Lab, and he is working on a novel algorithm to detect carcinogenic variants in RNA sequencing data. The gget opentargets module has many applications.

Bioinformatics

SugarCone Biotech

JULY 6, 2025

On this basis, and to drive use of the RNA sequencing technology that identifies NRG1 fusions, the drug was purchased by Partner Therapeutics. Drug development has also been hampered by serious adverse events, including Grade 3 or higher levels of neutropenia and interstitial lung disease. regulatory application in NSCLC.

Drug Development

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

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. This essay focuses on how we might do both, specifically for the cell. Subscribe to Asimov Press.

DNA

BioPharma Drive: Drug Pricing

JULY 16, 2025

On a conference call held Wednesday afternoon, Sarepta CEO Doug Ingram said that "failure to adapt" to recent setbacks "would risk our long-term viability as an organization and decrease the opportunity to bring the greatest benefit to the greatest number of patients living with rare disease."

Therapies

BioPharma Drive: Drug Pricing

JULY 10, 2025

Jacob Bell/BioPharma Dive Dive Brief: The Food and Drug Administration on Thursday granted full approval for Moderna’s COVID-19 vaccine Spikevax in children aged 6 months through 11 years who are at an increased risk for COVID disease. The shot was previously available for these individuals under emergency use authorization. Kennedy Jr.

Vaccine

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

Codon

AUGUST 4, 2025

This limitation stymies biology research, especially for diseases like Alzheimer’s, which are heavily influenced by post-translational modifications. Protein alterations, including phosphorylation and ubiquitination, can alter a protein’s function in ways that are invisible at the level of DNA or RNA.

Laboratories

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

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

Such clinical benefit is defined as a positive therapeutic effect that is clinically meaningful in the context of a given disease, is supported by a positive benefit-risk profile, and is, generally, a measure of how a patient feels, functions, or survives. in Duchenne Muscular Dystrophy).

FDA

Conversations in Drug Development Trends

APRIL 15, 2025

Early Phase Unit, Worldwide Clinical Trials Neurodegenerative diseases represent a diverse group of progressive disorders characterized by the loss of structure and function of neurons. Alzheimers Disease AD is a progressive neurodegenerative disorder characterized by cognitive decline and memory impairment.

Clinical Trials

BioPharma Drive: Drug Pricing

MAY 15, 2025

ADARx Pharmaceuticals will work with AbbVie to make RNA interference medicines for a variety of disease areas, including neurology, immunology and cancer.

RNA

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

Chemical Biology and Drug Design

JUNE 25, 2025

ABSTRACT Myocardial ischemia–reperfusion (MI/R) injury can lead to heart disease. ChIP, dual luciferase assay, and RNA pull-down were performed to explore the relation among KDM5A, miR-495-3p, and FOXO1. Then, the up-regulation of miR-495-3p negatively regulates the downstream FOXO1 in the cardiomyocyte cytoplasm.

RNA

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. The new method, called TRISCO, has the potential to transform our understanding of brain function, both in normal conditions and in disease, according to the new study.

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

Codon

JULY 21, 2025

There are only 5 nucleotide bases behind all life: adenine (A), cytosine (C), guanine (G) and thymine (T) in DNA, and uracil (U) taking the place of thymine in RNA. There are signs that what we currently call “rheumatoid arthritis” may turn out to be more than one disease. The same is true of biology.

Science

Science Daily: Pharmacology News

APRIL 9, 2025

The system can be formulated completely as RNA, dramatically simplifying delivery logistics compared to traditional systems that use both RNA and DNA. Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations.

RNA

BioPharma Drive: Drug Pricing

SEPTEMBER 19, 2023

AIRNA is one of several biotechs to debut plans for target alpha-1 antitrypsin deficiency in recent years, with competitors such as AlveoGene, Wave Life Sciences and Arrowhead Pharmaceuticals.

RNA

BioPharma Drive: Drug Pricing

SEPTEMBER 27, 2023

The Swiss drugmaker is restocking its pipeline by buying into preclinical Ionis medicines for Alzheimer’s and Huntington’s diseases.

RNA