Breakthrough Parkinson’s Drug Targets Disease at Its Genetic Roots

BIIB094, an antisense oligonucleotide for Parkinson’s disease, was well tolerated in an early randomized trial of 82 patients.

The drug lowered LRRK2 levels in cerebrospinal fluid by as much as 59%, showing the target can be suppressed safely in patients.

Researchers did not test whether the treatment improved symptoms or slowed disease progression, so clinical benefit remains unknown.

The results, published in Nature Medicine, support further trials of LRRK2-targeted gene-silencing therapy.