Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
AssigneesThe Regents of the University of Colorado, a body corporate, Travere Therapeutics Switzerland GmbH
InventorsErez Bublil, Frank Glavin, Marcia Sellos-Moura, Jan P. Kraus, Tomas Majtan, Randy Wanner, Orhan Causevic
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Abstract
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.
Claims22
A lyophilized formulation of a drug substance, wherein:
- (i) the drug substance comprises:
- (a) an isolated cystathionine B-synthase (CBS) protein comprising SEQ ID NO:
- 1;
- and
- (b) a polyethylene glycol (PEG) molecule covalently bound to the CBS protein;
- and
- (ii) wherein, upon reconstitution of the lyophilized formulation to a reconstituted liquid formulation, the reconstituted liquid formulation comprises:
- (a) the drug substance in a concentration of about 20-30 mg/ml;
- (b) about 15 mM of potassium phosphate;
- and
- (c) about 8% (w/v) trehalose.
A reconstituted liquid formulation comprising:
- (i) a drug substance in an amount of about 25 mg, wherein the drug substance comprises:
- (a) an isolated cystathionine β-synthase (CBS) protein comprising SEQ ID NO:
- 1;
- and
- (b) a PEG molecule covalently bound to the CBS protein;
- and
- (ii) about 15 mM of potassium phosphate;
- (iii) about 8% (w/v) trehalose;
- and
- (iv) 1 mL of water.
Dependencies are inferred from the claim text.