Congenital Muscular Dystrophy (CMD)
Delivers siRNA, antisense oligonucleotides, linear or circular mRNA and other RNA modalities to extrahepatic tissues - demonstrated across various indications in oncology (e.g. PDAC, breast cancer, ovarian cancer, lung cancer, melanoma, ATLL, TME), and inflammatory / autoimmune diseases (e.g. OA, RA, enterocolitis), CV (e.g. atherosclerosis, aortic aneurysm)
Siklos is indicated for the prevention of recurrent painful vaso-occlusive crises including acute chest syndrome in adults, adolescents and children older than 2 years suffering from symptomatic sickle cell syndrome
