公开(公告)号：US11939600B2

公开(公告)日：2024-03-26

申请号：US16617462

申请日：2018-05-31

Applicant: Arcturus Therapeutics, Inc.

Inventor： Kiyoshi Tachikawa ,  Carlos Gustavo Perez-Garcia ,  Padmanabh Chivukula ,  Hari Bhaskaran ,  Sean Christopher Daugherty ,  Christian W. Cobaugh

IPC: C12N9/02 ,  A61K31/7115 ,  A61K48/00

CPC classification number: C12N9/0071 ,  A61K31/7115 ,  A61K48/005 ,  C12Y114/16001 ,  A61K48/00

Abstract: This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.