RNA therapeutics company SiSaf has published positive safety and efficacy data for its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The data demonstrate that siRNA complexed with SiSaf’s silicon stabilised hybrid lipid nanoparticles (sshLNP) was able to significantly downregulate expression of an ADO2-specific mutant gene. The company says these results could have significant translational impact on […]
