AZ gets closer to Ionis, licensing amyloidosis drug in $3.6b...

AstraZeneca has snapped up another Ionis-discovered drug for its pipeline, this time phase 3 candidate eplontersen for the disease transthyretin amyloidosis (ATTR).

Alnylam launches digital tool for ATTR amyloidosis patients

Alnylam has been a pioneer in hereditary ATTR amyloidosis, getting approval in 2018 for the first drug to treat the rare disease, and has now launched a digital companion to help patients t

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

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis.

Intellia, Regeneron ace first trial with 'in vivo' CRISPR dr...

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening r

Vyndaqel romps away in US, spurring earnings hike from Pfize...

Alnylam gears up for first RNAi therapeutic filing in 2017

US biotech Alnylam says it could file its first product in 2017, a rare disease drug using what has long promised to be a revolutionary approach, RNA interference.