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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

The American Society of Clinical Oncology (ASCO) annual conference was one of the highlights of the year in cancer research and the fact it was held virtually did not distract from some stunning clinical lung cancer trial results presented by AstraZeneca. In the 500 patient trial, patients treated with Trodelvy lived for a median of 12.1

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Unfolding The Folds Of Transthyretin

Delveinsight

Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA.

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MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

pharmaphorum

Zilebesiran (formerly ALN-AGT) is one of a new breed of gene-silencing drugs that are intended to treat common, chronic diseases with infrequent dosing to boost compliance with treatment. In a phase 1 trial, zilebesiran was found to be well-tolerated and improve systolic blood pressure over 12 weeks with a single injection.

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Alnylam finds belly fat gene and drug target in UK Biobank study

pharmaphorum

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease.