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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. Zinc fingers. The genomic medicine journey.

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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. it can be reawakened.

Protein 52
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Mapping Networks of Immune Genes Behind Autoimmune Diseases

XTalks

For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. The proteins are interconnected and changes in one protein level can impact that of another.

Gene 98
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Double knockout strategy could boost CAR-T in solid tumours

Drug Discovery World

The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times greater T cell expansion in models, resulting in increased antitumour immune activity and durability. “We

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Advancements in upstream CLD for enhanced biotherapeutics production

Drug Discovery World

In addition, it has stimulated increased collaboration among drug developers, technology providers, supply chain organisations, and regulators. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics. Biotechnol Bioeng 2012;118:2326. Metab Eng 2020;57:203.

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Moderna broadens R&D scope with Vertex, Chiesi alliances

pharmaphorum

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

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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.