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)
Has the potential to treat multiple rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses, and Niemann-Pick disease type C (NPC).
Non-melanoma skin cancers and rare genetic skin manifestations.
