BioPharma Drive: Drug Pricing

JULY 17, 2023

For $500 million, Novartis will acquire DTx Pharma and its preclinical neurological disease drugs, marking the Swiss company’s latest investment in gene-silencing medicines.

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RNA Drugs Disease 246

BioPharma Drive: Drug Pricing

APRIL 1, 2025

Airna will use the Series B round to launch a Phase 1/2 trial of its lead program for alpha-1 antitrypsin deficiency, a disease targeted by several other firms.

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RNA Trials Disease 173

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.

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BioPharma Drive: Drug Pricing

NOVEMBER 28, 2023

Tuesday’s deal signals the pharmaceutical giant's confidence in Avidity Biosciences and its so-called antibody oligonucleotide conjugates.

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RNA Disease Pharmaceuticals Drugs 173

Chemical Biology and Drug Design

DECEMBER 13, 2023

This up-to-date commentary/perspective article sheds light on the revolutionary approaches designed to target the principal viral weapon “RNA” used by RNA viruses (e.g., the severe acute respiratory syndrome coronavirus 2 “SARS-CoV-2”) to infect humans and spread infections, the genomic RNA strands.

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RNA Disease Virus Drugs 100

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.

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RNA Small Molecule Disease Assay Development 96

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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RNA Research Therapies Disease 183

SCIENMAG: Medicine & Health

JULY 19, 2023

Together, they have published dozens of papers on the mechanics of mitochondrial DNA and RNA in a single-celled, disease-causing parasite called Trypanosoma brucei. As molecular biologists at Boston University and as husband and wife, Ruslan Afasizhev and Inna Afasizheva, have worked together for decades.

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RNA Disease DNA 83

SCIENMAG: Medicine & Health

AUGUST 14, 2023

A technique that enables scientists to record gene mutations and patterns of gene activity in individual cells has been extended to cover RNA splicing as well, in a study led by researchers at Weill Cornell Medicine, the New York Genome Center and the Princess Margaret Cancer Centre in Toronto.

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RNA Disease Research 83

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.

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Research RNA Medical Schools Treatment 127

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

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BioPharma Drive: Drug Pricing

AUGUST 9, 2023

The Flagship-backed company claims its technology can create genetic drugs that work broadly to treat diseases caused by “stop” mutations.

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RNA Drugs Disease 130

Drug Target Review

JANUARY 24, 2024

Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). Alzheimer’s disease Alzheimer’s is the most common cause of dementia. 4 Short RNAs (sRNAs) do not code for proteins.

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RNA Disease DNA Engineering 64

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.

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RNA Science Research Disease 99

BioPharma Drive: Drug Pricing

AUGUST 3, 2023

The biotech is acquiring rights to an RNA interference medicine Alnylam invented for a rare blood disorder, continuing a corporate makeover that began in 2020.

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RNA Disease 130

Broad Institute

SEPTEMBER 28, 2023

By Allessandra DiCorato September 28, 2023 Credit: Strittmatter Laboratory, Yale University Amyloid beta clusters (red) builds up among neurons (green) in a mouse model of Alzheimer’s disease. The scientists identified a suite of changes in cells unique to the early stages of Alzheimer’s, including some not seen before in animal studies.

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Disease Medical Schools Research Hospitals 144

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 147

Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease?

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Therapies Disease RNA Engineering 102

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

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Therapies Disease Engineering Medical Schools 116

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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Therapies Disease Treatment RNA 98

ASPET

SEPTEMBER 6, 2023

Toll-like receptor (TLR) 7 and TLR8 are single-stranded RNA-sensing endosomal pattern recognition receptors that evolved to defend against viral infections. However, aberrant TLR7/8 activation by endogenous ligands has been implicated in the pathogenesis of autoimmune diseases including systemic lupus erythematosus.

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Disease RNA Treatment Regulations 100

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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RNA Immune Response Therapies Disease 128

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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RNA Bioinformatics Science Pharmacokinetics 59

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

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.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. Figure 1: High-level workflow for early drug discovery Once the raw data has been gathered, the next step is to gain a thorough understanding of the data.

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RNA Drugs Research Science 52

The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

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RNA Small Molecule DNA Licensing 52

Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. By Sarah C.P.

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Engineering Research Virus DNA 144

Broad Institute

JUNE 18, 2024

By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples. Tags: Infectious Disease Diagnostics Pardis Sabeti Paper cited Zhang YB, Arizti-Sanz J, et al. 2024.04.004.

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Virus RNA Laboratories Medical Schools 144

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

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

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DNA RNA Disease Hospitals 128

Broad Institute

APRIL 4, 2024

A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis By Rose Circeo April 4, 2024 Breadcrumb Home A Practical guide to bulk RNA-seq, including guidelines for eQTL analysis The Primer on Medical and Population Genetics is a series of weekly lectures on genetics topics related to human populations and disease.

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RNA Bioethics Disease Research 52

Broad Institute

DECEMBER 6, 2023

Cytokines are also targeted by drugs for many diseases such as rheumatoid arthritis, COVID-19, and cancer, but until now, scientists haven’t had a comprehensive view of how different immune cells respond to different cytokines because the immune system is so complex. For many immune-mediated diseases, there's no cure or treatment.

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Immune Response RNA Disease Medical Schools 138

Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.

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RNA Medical Schools Engineering Disease 98

ASPET

AUGUST 10, 2023

In this study, using RNA interference library screening, downstream anti-apoptotic molecular signaling components involved in CA-induced STC2-mediated protection against ethanol-induced apoptosis were investigated.

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RNA Regulations Treatment Disease 100

Broad Institute

NOVEMBER 23, 2023

The scientists found a surprising number and diversity of CRISPR systems, including ones that could make edits to DNA in human cells, others that can target RNA, and many with a variety of other functions. The new systems could potentially be harnessed to edit mammalian cells with fewer off-target effects than current Cas9 systems.

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RNA DNA Engineering Science 135

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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