Posted by Michael Wonder on 16 May 2023
FDA grants SiSaf’s innovative siRNA therapy SIS-101-ADO orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis
15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis by the US FDA.
SIS-101-ADO combines an siRNA that suppresses the expression of CLCN7 with SiSaf’s Bio-Courier delivery technology.
