SiSaf exercises its option under licencing agreement on gene therapy for rare bone disease and announces progress on IND enabling studies

Guildford, UK, 4 November 2021 – SiSaf Ltd, a company developing RNA therapeutics for rare genetic skeletal disorders, today announced that it has exercised its option under its collaboration with the University of L’Aquila, Italy. The license enables SiSaf to develop small interfering RNA (siRNA) targeted via its Bio-Courier® next generation silicon stabilized hybrid lipid nanoparticles (sshLNP) to regulate the expression of a mutant gene expressed by osteoclasts and other cell types responsible for causing the therapeutically neglected rare autosomal dominant disorder type 2 Osteopetrosis (ADO2) in adults.

Read the full release here.