Delveinsight
AUGUST 6, 2020
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.
pharmaphorum
DECEMBER 23, 2020
The company’s menopause drug NT-814 is heading for phase 3 development in 2021 following a mid-stage trial readout. 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. Rare disease progress.
pharmaphorum
JULY 28, 2022
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.
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