Ionis doses ALS patients with antisense medicine in clinical trial

By The Science Advisory Board staff writers

October 22, 2020 -- Ionis Pharmaceuticals has dosed the first patients with ION541, an investigational antisense medicine developed to treat most forms of amyotrophic lateral sclerosis (ALS), in a phase I/II clinical trial.

ALS is a rare, progress neurodegenerative disorder that affects approximately 55,000 people globally. Almost all cases of ALS display TDP-43 protein aggregation in motor neurons. ION541 targets ataxin-2 RNA (ATXN2), which has been shown to prevent or reverse TDP-43 toxicity in preclinical models of ALS.

Ionis received a payment of $10 million from Biogen for the initiation of the phase I/II clinical trial of ION541. Biogen is developing ION541 as part of a broad collaboration with Ionis to advance novel antisense therapies for the treatment of neurological disorders.

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