Remove Antibody Remove Clinical Trials Remove Gene Remove Gene Silencing
article thumbnail

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a life-threatening disease affecting the heart and nervous system. All told, the deal could be worth up to $1.2

article thumbnail

Cash round sets up trial of InteRNA’s microRNA for cancer

pharmaphorum

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 59
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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

pharmaphorum

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.”. The antibodies produced as a result lodge in the kidneys, causing severe complications.

article thumbnail

Unfolding The Folds Of Transthyretin

Delveinsight

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 Therapies. ATTR amyloidosis can be either hereditary or acquired (non-hereditary). hATTR market : In The Pipeline.

Protein 52