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Conversations from ESMO Targeted Anticancer Therapies Congress 2024 

Drug Discovery World

Additional approaches, such as profiling of RNA and proteins, and the use of artificial intelligence (AI), could be key to providing a more comprehensive picture of a patient’s tumour and enable the use of optimised treatments. MT: Why was STORM attending the conference? We were there to present the latest findings from our research.

RNA 64
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FDA-approved drug sensitises brain cancer cells to radiotherapy

Drug Discovery World

This action sensitised GBM cells to radiotherapy both in vitro and in vivo (in mouse models). “Our research has revealed cladribine as a radiosensitiser for GBM treatment by drug repurposing, which can offer multiple advantages,” says Prof Youn. “As GBM is a WHO grade IV brain tumour with dismal prognosis.

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Taking an ‘upside-down’ approach to mRNA delivery

pharmaphorum

The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential.

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Receptor Occupancy Assays by Flow Cytometry: Benefits for Clinical Trials

XTalks

Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. What is Flow Cytometry?

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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. This method is generally used to deliver small interfering RNA (siRNA), but it can also be used for larger oligonucleotides too. Oligonucleotide. Anti-miRNA.

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What the Glycome Can Tell Us About Persistent HIV Infection

XTalks

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

RNA 52
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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. GeneTx and Ultragenyx are co-developing the therapy.