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AMO's CNS ammunition

AMO is repurposing compounds to treat CNS deficits in fragile X and DM1

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.

AMO licensed AMO-01 from Thallion Pharmaceuticals Inc. (now part of Bellus Health Inc.). Thallion had discontinued a Phase II trial of AMO-01 in glioblastoma multiforme (GBM) due to lack

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