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RBr054 intrathecal for neurodegenerative disease​

RBr054 is an RNA therapeutic, a 'splice-switching oligonucleotide' or SSO, designed to increase esRAGE levels in the brain and CNS. esRAGE is a naturally occurring, anti-inflammatory spliceoform of RAGE.


RBr054 is under investigation for neurodegenerative disease including Alzheimer's disease.


RBr054 is an intrathecal injection designed to switch the equilibrium back towards healthy levels of esRAGE in the brain and CNS, inhibit pro-inflammatory RAGE signalling in areas of neurodegeneration, and protect against further damage.