Patent classifications
C12Y402/01022
CHEMICALLY MODIFIED CYSTATHIONINE BETA-SYNTHASE ENZYME FOR TREATMENT OF HOMOCYSTINURIA
The invention provides reagents and methods for enzyme replacement therapy using chemically modified species of human cystathionine -synthase (CBS) to treat homocystinuria and other related diseases and disorders.
CELLULAR REPROGRAMMING BY TARGETING THE GOLGI APPARATUS
The present invention generally relates to methods and compositions for the generation of T cells with enhanced features and enhanced therapeutic properties. The present invention also relates to methods of treating or preventing cancer with the T cells which display enhanced features and enhanced therapeutic properties.
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 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.
CYSTEINE PROTOTROPHY
Provided herein are cells, compositions, and related methods for improved cell growth. The expression of one or more of CBS, CTH, and GNMT genes is increased. In some embodiments, nucleic acid constructs, vectors, host cells and related compositions and methods for generating and selecting cysteine prototroph cells are provided.