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Unlocking the potential of mRNA for the future treatment of rare diseases 

Drug Discovery World

This presents a unique opportunity for mRNA-based therapies to potentially restore normal cell function by supplying copies of the functional transcript for the cell to read. mRNA enables the delivery of a transiently expressed genetic molecule that is translated into a target protein using the machinery of the host cell.

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The path to improved safety of gene-based products

Drug Discovery World

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Host cell proteins (HCPs) – proteins derived from the host cells used for viral production—are one class of process-related impurities.

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Improving quality control for CAR T cell therapies

Drug Discovery World

To overcome this evasion tactic, the CAR protein was developed to recognise other markers on cancer cells. CAR is a fusion protein composed of an extracellular antibody fragment that recognises antigens and an intracellular domain that directs the T cell to the tumour. Identifying the right CAR T cell target.

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The Utility of Liquid Biopsy in Oncology Clinical Trials

XTalks

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. However, Dr. Bahassi reported that the selection of CTCs using this method is often not very efficient and the CTC population is usually highly contaminated with leukocytes.

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RRx-001: Jack of all trades, master of…many

Drug Discovery World

RRx-001 directly and irreversibly binds to a specific amino acid cysteine residue on a protein complex called the NLRP3 inflammasome, which serves as the “on switch” for inflammation.

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COVID-19 Pandemic Coverage

XTalks

The company’s candidate vaccine, mRNA-1273, is a synthetic messenger RNA that encodes the stabilized SARS-CoV-2 spike protein. The University of Oxford/AstraZeneca partnership, in turn, is testing a viral-vectored coronavirus vaccine that again expresses the spike protein of SARS-CoV-2 virus.