[{"orgOrder":0,"company":"SiSaf","sponsor":"CSSi LifeSciences","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SiSaf's Innovative RNA Therapeutic for Rare Genetic Skeletal Disorders Begins the U.S. Regulatory Process for Orphan Drug Designation","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"IND Enabling","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"IND Enabling"},{"orgOrder":0,"company":"SiSaf","sponsor":"University of Leipzig","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"SiSaf and the University of Leipzig Partner to Develop Bio-Courier Targeted miRNA for the Treatment of Pancreatic Cancer","therapeuticArea":"Oncology","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Oncology","graph2":"Preclinical"},{"orgOrder":0,"company":"SiSaf","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants SiSaf\u2019s Innovative siRNA Therapy SIS-101-ADO Orphan Drug Designation and Rare Pediatric Disease Designation for the Treatment of Autosomal Dominant Osteopetrosis","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"IND Enabling","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"IND Enabling"}]
Find Clinical Drug Pipeline Developments & Deals by SiSaf
SIS-101-ADO is an siRNA that suppresses the expression of CLCN7, a mutant gene expressed by osteoclasts and other cell types responsible for causing ADO2, the RNA therapy restores bone mass and quality to nearly normal levels.
The collaboration will combine SiSaf’s expertise in RNA delivery using its Bio-Courier sshLNPs and the University of Leipzig’s expertise in miRNA targeting and therapeutic approaches in cancer, with an initial focus on pancreatic cancer to develop miRNA-based Therapies.
SIS-101-ADO combines an siRNA that suppress expression of CLCN7, with SiSaf’s Bio-Courier® next generation silicon stabilized hybrid lipid nanoparticles technology of other RNA delivery technologies by merging LNP technology with inorganic bioabsorbable silicon.