Positive preclinical data for siRNA therapy for osteopetrosis
Drug Discovery World
SEPTEMBER 25, 2023
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 bone disease therapies and open the path to human trials of it’s potentiall
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