Proqr hopes to usherin a new ophthalmic therapy
The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
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.
Wave gets another shot at Huntington’s
The group hopes for a better result with a higher dose of its antisense projects, but it also has a back-up plan.
Translate’s investment case shifts fully to Covid-19
The company’s lead cystic fibrosis asset fails, shifting the focus to the Sanofi-partnered Covid-19 vaccine.
Ovid crashes out in Angelman syndrome
The failure of OV101 puts the spotlight on Ultragenyx and Roche.
Ionis joins the inhaled cystic fibrosis therapy race
Ionis’s inhaled antisense candidate shows promising signs, with Arrowhead and Translate Bio also in the running.
