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

RNA 64

RNA Small Molecule Disease Research 64

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.

RNA 52

RNA DNA Vaccine Drug Development 52

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.

Disease 142

Disease Therapies Engineering Clinical Trials 142

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.

RNA 52

RNA Bioethics Disease Research 52

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?

Therapies 102

Therapies Disease RNA Engineering 102

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 122

RNA Disease Science Pharmaceuticals 122

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.

Therapies 114

Therapies Disease Engineering Medical Schools 114

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.

Disease 100

Disease RNA Treatment Regulations 100

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 117

RNA Disease 117

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?

Therapies 98

Therapies Disease Treatment RNA 98

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.

RNA 64

RNA Disease Pharmaceuticals Drugs 64

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.

Disease 144

Disease Medical Schools Research Hospitals 144

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.

RNA 52

RNA Small Molecule DNA Licensing 52

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.

RNA 98

RNA Drugs Disease 98

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 128

RNA Immune Response Therapies Disease 128

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., SCHEME PATENT PTC Therapeutics Inc.,

Small Molecule 57

Small Molecule RNA DNA Disease 57

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. In fact, it was these early pharmaceutical successes that gave us the confidence that we would ultimately succeed in systematically drugging a wide range of RNA structures.

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

Crown Bioscience

JULY 15, 2024

Precision medicine approaches rely on biomarkers to define optimal treatment strategies and also play important roles in the diagnosis of disease, treatment efficacy assessment, and/or disease progression.

RNA 52

RNA Therapies Treatment Disease 52

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 100

RNA Virus Regulations Disease 100

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.

RNA 52

RNA Drugs Disease 52

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.

DNA 128

DNA RNA Disease Hospitals 128

ASPET

OCTOBER 8, 2024

However, the disease eventually progresses as castration-resistant PCa (CRPC). Finally, RNA sequencing revealed a potential link in the downregulation of Ras/MAPK signaling following combination treatment. Androgen deprivation is the standard treatment for prostate cancer (PCa) patients.

RNA 100

RNA Treatment Therapies Disease 100

Reprocell

AUGUST 8, 2024

In the ever-evolving landscape of drug discovery, the ability to extract and analyze DNA and RNA from clinical biospecimens has become a cornerstone of innovative research. As we delve deeper into the molecular mechanisms of diseases, the quality of nucleic acid extraction plays a critical role in unlocking new therapeutic avenues.

Clinical Research 52

Clinical Research Research RNA DNA 52

Drug Target Review

JULY 11, 2023

In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.

Disease 75

Disease RNA Small Molecule Clinical Development 75

Broad Institute

OCTOBER 21, 2024

The method, called Genotypic and Phenotypic Antibiotic Susceptibility Testing through RNA detection, or GoPhAST-R, analyzes the growth and genetic activity of the bacteria to quickly determine the pathogen’s susceptibility to various medicines.

RNA 115

RNA Hospitals Laboratories Medical Schools 115

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.

RNA 100

RNA Regulations Treatment Disease 100

Chemical Biology and Drug Design

NOVEMBER 13, 2023

SARS-CoV-2 is responsible for the coronavirus disease of 2019 (COVID-19) which is one of the most widespread and powerful infections affecting human lungs. WHO proclaimed the outbreak of the Ebola virus disease (EVD), in 2014 that killed hundreds of people in West Africa.

Vaccine 100

Vaccine Laboratories RNA Clinical Development 100

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.

RNA 52

RNA Disease 52

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.

RNA 54

RNA Research Therapies Disease 54

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.

Engineering 144

Engineering Research Virus DNA 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.

Science 56

Science RNA Therapies Disease 56

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.

RNA 84

RNA Virus DNA Vaccine 84

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.

RNA 98

RNA Medical Schools Engineering DNA 98

PLOS: DNA Science

JULY 27, 2023

That information led, thanks to vaccine shelved from the first SARS circa 2003, to the rapid development and deployment of mRNA vaccines against the new infectious disease. Seventeen of the entire group (10.7%) had “unanticipated monogenic disease risks (uMDRs).” But, they have genomes, built of RNA or DNA.

Disease 98

Disease DNA Virus RNA 98

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 127

Research RNA Medical Schools Treatment 127

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.

Virus 144

Virus RNA Laboratories Medical Schools 144

Chemical Biology and Drug Design

JULY 27, 2023

cells induced by poly (I:C) were treated with or without PA, and then RNA-sequencing, GO and KEGG analysis were carried out to obtain the five differentially expressed genes (IL-1β, HMOX1, PTGS2, Abca1, and P65), which were verified by QRT-PCR and western blot. RNA-sequencing was applied to analyze the differentially expressed genes (DEGs).

RNA 100

RNA Virus Disease Drugs 100