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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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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.

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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

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