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New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing

pharmaphorum

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. fundraiser in July for the next stage of its development. million compared with £3.8

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UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

Developed by Orchard Therapeutics, Libmeldy received approval from the European Commission (EC) in December 2020 and about a month later, received a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) to expedite its path to approval in the US. million (approximately $3.8

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NIH renews knockout mouse project for 3rd time

Drug Discovery World

Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. . Institutions in 15 countries.

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

DDW Editor Reece Armstrong explores some of the trends that will be featured in sessions throughout ELRIG Drug Discovery 2022 and the news behind them. With 3,633 advanced therapeutical medicinal products (ATMPs) currently in some form of clinical or pre- clinical development, the sector shows no signs of slowing down. Automation.

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World RNA Day: What impact RNA it had on drug discovery?  

Drug Discovery World

The NIH states: “This natural mechanism for sequence-specific gene silencing promises to revolutionise experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.” There are many examples of ways in which RNAi is impacting drug discovery.

RNA 52
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The use of base editing in stem-cell based therapies

Drug Discovery World

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.