RAB-008c

RAB-008c is a preclinical small molecule candidate developed by RabPharma for the treatment of Fibrous Dysplasia (FD). FD is a rare skeletal disorder caused by somatic activating mutations in the *GNAS* gene, which encodes the G-protein subunit alpha S (Gsα). These mutations lead to the constitutive activation of adenylyl cyclase and the subsequent overproduction of cyclic AMP (cAMP), which triggers the replacement of normal bone and marrow with fibrous-osseous tissue. RAB-008c is designed as a targeted inhibitor of the Gsα/cAMP signaling pathway. It is formulated for intra-articular administration, aiming to provide localized therapeutic effects for bone lesions and associated pain in patients with FD.