PEGYLATED CYSTATHIONINE BETA SYNTHASE FOR ENZYME THERAPY FOR TREATMENT OF HOMOCYSTINURIA

The present disclosure provides formulations for a drug product comprising a PEGylated CBS protein having the amino acid sequence of SEQ ID NO: 1. Dosages and dosing regimens are provided for treatment of homocystinuria in a subject in need thereof. Additionally, the dosages and dosing regimens are...

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Bibliographic Details
Main Authors WANNER, Randy, SELLOS-MOURA, Marcia, MAJTAN, Tomas, CAUSEVIC, Orhan, KRAUS, Jan P, GLAVIN, Frank, BUBLIL, Erez
Format Patent
LanguageEnglish
French
German
Published 04.05.2022
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:The present disclosure provides formulations for a drug product comprising a PEGylated CBS protein having the amino acid sequence of SEQ ID NO: 1. Dosages and dosing regimens are provided for treatment of homocystinuria in a subject in need thereof. Additionally, the dosages and dosing regimens are also provided to reduce the level of homocysteine (Hcy) or increase the levels of cysteine (Cys) and/or cystathionine (Cth) in a subject in need thereof.
Bibliography:Application Number: EP20200743019