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Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. The exact mechanism depends on the disease in question.

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This week in drug discovery (2-6 October)  

Drug Discovery World

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52
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Gene Therapy and Pharmacokinetics

Camargo

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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The Dose: DDW’s drug discovery highlights

Drug Discovery World

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month. . Everyone enjoys a good news story and the drug discovery and development sector is no exception. The post The Dose: DDW’s drug discovery highlights appeared first on Drug Discovery World (DDW).

Drugs 52
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Right on target: The shift to precision pharmaceuticals

Drug Discovery World

If there’s one thing I would take from the Summer 2023 issue of Drug Discovery World , it would be that the application of technologies and processes throughout the pharmaceutical industry are changing how scientists pursue the development of therapies. This was done to assess the accuracy of the team’s machine-learning algorithms.

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Epigenetic Editing with CRISPR + ADHD in Preschoolers – Xtalks Life Science Podcast Ep. 10

XTalks

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

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Delivering on the promise of gene editing

Drug Discovery World

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.