SiSaf’s Innovative RNA Therapeutic for Rare Genetic Skeletal Disorders Begins the U.S. Regulatory Process for Orphan Drug Designation

GUILDFORD, England, 15 MAY 2023: SiSaf Ltd, an RNA delivery and therapeutics company, announces that SIS-101-ADO, its siRNA therapeutic for patients with Autosomal Dominant Osteopetrosis Type 2 (ADO2), has been granted Orphan Drug Designation by the U.S. FDA.

In addition, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the treatment of Autosomal Dominant Osteopetrosis.