neurodevelopmental disorders
AstraZeneca/Ionis’ Wainzua granted EC approval to treat rare disease ATTRv-PN
ATTRv with polyneuropathy affects up to 40,000 people worldwide
AstraZeneca receives MHRA approval for Wainzua in rare nerve disease ATTRv-PN
The therapy is designed to reduce the effects of the disease by lowering the production of TTR protein
Ionis’ investigational antisense therapy shows promise in Angelman syndrome
ION582 is expected to enter late-stage development for the rare neurodevelopmental disorder in 2025
Ionis shares positive late-stage results for olezarsen in familial chylomicronaemia syndrome
The rare genetic disease is estimated to affect up to 13 people per million in the US
Ionis shares positive phase 3 results for hereditary angioedema drug candidate
More than 20,000 people in the US and Europe are affected by the rare genetic disease
Ionis reports positive phase 2b results for fesomersen in thrombosis prevention
The company also announced it will regain rights to the antisense medicine from Bayer
AstraZeneca and Ionis sign $3.6bn deal for eplontersen
The liver-targeted antisense therapy is in phase 3 trials for the treatment of transthyretin amyloidosis, a systemic, progressive and fatal condition
GSK’s Ionis-partnered hepatitis B drug hits the mark in phase 2
Treatment could be transformative for around 260 million people living with disease