Invention Grant
- Patent Title: Compositions and methods for treating phenylketonuria
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Application No.: US16617462Application Date: 2018-05-31
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Publication No.: US11939600B2Publication Date: 2024-03-26
- Inventor: Kiyoshi Tachikawa , Carlos Gustavo Perez-Garcia , Padmanabh Chivukula , Hari Bhaskaran , Sean Christopher Daugherty , Christian W. Cobaugh
- Applicant: Arcturus Therapeutics, Inc.
- Applicant Address: US CA San Diego
- Assignee: Arcturus Therapeutics, Inc.
- Current Assignee: Arcturus Therapeutics, Inc.
- Current Assignee Address: US CA San Diego
- Agency: MINTZ, LEVIN, COHN, FERRIS, GLOVSKY AND POPEO, P.C.
- International Application: PCT/US2018/035476 2018.05.31
- International Announcement: WO2018/222925A 2018.12.06
- Date entered country: 2019-11-26
- Main IPC: C12N9/02
- IPC: C12N9/02 ; A61K31/7115 ; 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.
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