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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Currently, only a small number of CPP-based oligonucleotide delivery examples have been reported for in vivo application. In a recent publication from scientists at the University of Bordeaux, a CPP-based nanoparticles approach was reportedly used to deliver siRNA into cancer cells of solid tumours. Covalent (thiol-maleimide) 7.

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Fibrosis research: Advances and challenges 

Drug Discovery World

Fibrocor is on track to complete all IND-enabling work to support IND filing and initiation of clinical trials in 2024. Phase I of the clinical program will dose healthy volunteers with single ascending doses of FIB992 before proceeding to a multiple ascending dose study where the drug is administered for a two-week period.

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FAIR Game: making data work harder in the race to market 

Drug Discovery World

For pharma and biotech companies – whether working on small or large molecule drugs, or cell and gene therapies – this means empowering scientists with better and faster data management approaches, best practices, and leading technologies so they can pinpoint new molecules and formulations and modify existing ones more easily and quickly.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing.

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Synthetic biology tools advancing and accelerating drug discovery efforts

Drug Discovery World

Even as methods in DNA synthesis continue to advance, scientists’ reliance on third-party companies for synthesising DNA creates a bottleneck, impeding research timelines. . “If If you want to order gene-length sequences, rather than short oligos for PCR, the waiting times are around two or three weeks at least.

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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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.