SiSaf

siRNA
#Bone disease #Preclinical investigations #RNA #siRNA #SiSaf

Positive preclinical data for siRNA therapy for osteopetrosis

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. Read More

#News

siRNA
#FDA #Orphan drugs #Paediatrics #Rare disease #siRNA #SiSaf

Therapy for rare skeletal disorder granted orphan status

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to SiSaf’s small interfering RNA (siRNA) therapeutic for skeletal disorder Autosomal Dominant Osteopetrosis Type 2 (ADO2). In addition, due to the serious manifestations of this rare disease in children, SIS-101-ADO has been granted Rare… Read More

#News #Regulatory

Pancreatic cancer cells
#Cancer research #Nanomedicine / nanotechnology #Oncology #Pancreatic cancer #RNA #SiSaf

Partnership will develop targeted miRNAs to treat pancreatic cancer

SiSaf is to collaborate with the University of Leipzig, Germany, to develop targeted micro interfering RNAs (miRNA) for the treatment of cancer, with an initial focus on pancreatic cancer.   The collaboration will combine SiSaf’s expertise in RNA delivery using its Bio-Courier silicon-stabilised hybrid lipid nanoparticles (sshLNPs) and… Read More

#Cancer Research #News