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. Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one.

Gene Silencing 52

Gene Silencing Protein Marketing Gene 52

pharmaphorum

DECEMBER 23, 2020

2020 also saw some of the first “tumour agnostic” cancer drugs get to market, with Bayer’s Vitravki (larotrectinib) getting funding in the UK for tumours with confirmed neurotrophic tyrosine receptor kinase (NTRK) gene fusions.”. Rare disease progress.

Marketing 76

Marketing Gene Silencing Gene Drugs 76

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. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

pharmaphorum

JUNE 8, 2022

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.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52