Japan, Feb. 24 -- TRANSLATE BIO INC has got intellectual property rights for 'MRNA THERAPY FOR ARGINOSUCCINATE SYNTHETASE DEFICIENCY.' Other related details are as follows:
Application Number: JP,2021-212128
Category (FI): A61K47/24,C12N15/52@Z,C12N15/12,A61K38/43,A61K47/22,A61K47/28,A61K9/127,A61P43/00,111,A61P3/00,A61K48/00
Stage: PROBLEM TO BE SOLVED: To provide improved methods and compositions for treating arginosuccinate deficiency (ASD) based on mRNA therapy.SOLUTION: The invention provides methods and compositions for treating arginosuccinate synthetase deficiency (ASD) based on mRNA therapy. More specifically, the invention provides a method for treating ASD by administering to a subject in need of treatment a composition comprising a mRNA encoding arginosuccinate synthetase (ASS) at an effective dose and at an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in one or more liposomes. Liposomes suitable for the invention comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.SELECTED DRAWING: None (Grant)
Filing Date: Dec. 27, 2021
Publication Date: Feb. 18, 2022
The original document can be viewed at: https://www.j-platpat.inpit.go.jp/p0100
Disclaimer: Curated by HT Syndication.
