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

pharmaphorum

NT-814 is a non-hormonal drug that works on the neurokinin (NK) 1,3 receptor antagonist, thus treating the vasomotor symptoms associated with the menopause. The company’s menopause drug NT-814 is heading for phase 3 development in 2021 following a mid-stage trial readout. Rare disease progress.

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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.

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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.