SOM Biotech | Evaluate

September 06, 2021

Another gene therapy safety scare hits

The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.

Novo bets against Crispr for amyloidosis

Novo moves into amyloidosis not long after Intellia made a splash here.

March 23, 2021

The Huntington’s pipeline takes a blow

The failure of Roche and Ionis’s late-stage candidate tominersen casts doubt on Wave’s approach, too.

April 04, 2018

Therapy focus – Pfizer blows hereditary amyloidosis fight open

August 03, 2016

Therapy focus – One step forward but two steps back in Huntington’s

June 23, 2016

Therapy focus – Amyloidosis pipeline still looks to Alnylam for answers