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

pharmaphorum

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105
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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

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. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,