BioCentury
ARTICLE | Translation in Brief

Attackin' Ataxin

One antisense therapy for ALS and SCA2?

May 11, 2017 8:32 PM UTC

Two independent academic groups have shown different antisense oligos (ASOs) made by Ionis Pharmaceuticals Inc. (NASDAQ:IONS) targeting the same protein, ataxin 2 (ATXN2), extended survival and improved motor function in models of amyotrophic lateral sclerosis (ALS) and spinocerebellar ataxia type 2 (SCA2).

ATXN2 was identified roughly 25 years ago as the cause of SCA2, a disease in which cerebellar neurons degenerate leading to serious gait and upper body coordination problems. ...

BCIQ Target Profiles

Ataxin 2 (ATXN2) (SCA2)