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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. Cas9 binds and uses RNA as an address-tag.

Protein 52
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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. Experts speak DDW asked a range of drug discovery experts where they thought the drug discovery expertise happening in the UK.

Drugs 75
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Casting aside CRISPR scissors and making a point with base editors

pharmaphorum

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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The doors CRISPR libraries have and will open in phenotypic drug screening 

Drug Discovery World

The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. Bacterial RNA is replaced by a synthetic guide RNA (gRNA) that can recognise a specific target DNA sequence. CRISPR libraries: Why do we need them?

Genome 52
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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

DNA 98
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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.

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Reflecting on PEGS Europe 2023 

Drug Discovery World

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It The next presentation attended in this track was the keynote presentation, which was given by Kate Smith, PhD, Head of UK Protein & Cell Sciences at GSK. GSK has developed high-throughput mammalian and E.