Pipeline

We take aim at the world’s most intractable diseases. Our portfolio spans internal as well as partnered programs, across both novel and known (but traditionally undruggable) targets.

Indication

rModality

Target

Discovery

Pre-clinical

Clinical

Undisclosed

ALS

Proprietary

Cancer

Cancer

SM

SM

SM

SM

SM

ASO

Proprietary

FTD/AD

ALS

Proprietary

SM

SM

Proprietary

FTD/AD

FTD/AD

Undisclosed

Proprietary

Proprietary

ALS

SM = small molecule
rModality = RNA-targeted modality