Abstract
Methods and compositions for the transamniotic delivery of mRNA encoding alpha-1 antitrypsin protein to a fetus for the prenatal treatment of alpha-1 antitrypsin deficiency.
Full Text
What is claimed is:
Methods and compositions for the transamniotic delivery of mRNA encoding alpha-1 antitrypsin protein to a fetus for the prenatal treatment of alpha-1 antitrypsin deficiency.
Timeline
Filed
04/15/2026Published
08/20/2026Granted
Not AvailableIPC Codes(7)
A61K 31/7105:Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 9/00:Medicinal preparations characterised by special physical form
A61K 9/1272:comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers (lipids as modifying agents A61K 47/54)