ARTICLE | Emerging Company Profile
AMO's CNS ammunition
AMO is repurposing compounds to treat CNS deficits in fragile X and DM1
March 14, 2016 7:00 AM UTC
AMO Pharma Ltd. is in-licensing therapies for rare genetic diseases with no treatment options, starting with two potentially disease-modifying candidates for fragile X syndrome and myotonic dystrophy type 1.
The company's first candidate is AMO-01, a farnesylated dibenzodiazepinone in development for fragile X. AMO-01 targets the peripheral benzodiazepine receptor (TSPO; PBR), expressed on activated astrocytes, and inhibits the Ras-ERK pathway...