Lentiviral vector formulations
AssigneesBioverativ Therapeutics Inc., Fondazione Telethon, Ospedale San Raffaele S.R.L.
InventorsAndrew Kroetsch, Isidro Zarraga
Life of this patent
Estimating the term…
Abstract
Lentiviral vector (LV) formulations, and pharmaceutical compositions comprising such LV formulations, with improved stability and suitable for systemic administration are provided. Methods for treating disorders, especially blood disorders, using systemic administration of LV formulations are also provided.
Claims16
1-48. (canceled)
A reduced in-vivo toxicity recombinant lentiviral particle preparation comprising:
- (a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
- (b) a TRIS-free buffer system comprising a phosphate buffer;
- (c) a salt;
- (d) a poloxamer 188 (P188);
- (e) a carbohydrate;
- wherein the lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
- wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO:
- 3;
- wherein the recombinant lentiviral particle preparation is suitable for systemic administration to a human patient;
- and
- wherein systemic administration of the recombinant lentiviral particle preparation to the patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.
A reduced in-vivo toxicity recombinant lentiviral particle preparation, comprising:
- (a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
- (b) a TRIS-free buffer system comprising about 10 mM phosphate buffer;
- (c) about 100 mM or about 130 mM sodium chloride;
- (d) about 0.05% (w/v) P188;
- (e) about 3% (w/v) sucrose;
- wherein the pH of the preparation is about 7.3;
- wherein the lentiviral particle preparation is suitable for systemic administration to a human patient;
- wherein the recombinant lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
- wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO:
- 3;
- and
- wherein administration of the lentiviral particle preparation to a patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.
A reduced in-vivo toxicity recombinant lentiviral particle preparation, comprising:
- (a) a therapeutically effective dose of a lentiviral particle comprising a recombinant lentiviral vector;
- (b) a TRIS-free buffer system comprising about 10 mM phosphate buffer;
- (c) about 100 mM or about 130 mM sodium chloride;
- (d) about 0.05% (w/v) P188;
- (e) about 3% (w/v) sucrose;
- wherein the pH of the preparation is about 7.0;
- and wherein the lentiviral particle preparation is suitable for systemic administration to a human patient;
- wherein the recombinant lentiviral particle further comprises an envelope comprising a vesicular stomatitis virus G (VSV-G) protein or a fragment thereof;
- wherein the recombinant lentiviral vector comprises a nucleic acid comprising a nucleotide sequence that is at least 80% identical to a Factor IX (FIX) coding sequence set forth in SEQ ID NO:
- 3;
- and
- wherein administration of the lentiviral particle preparation to a patient has reduced in-vivo toxicity relative to a recombinant lentiviral particle preparation comprising a TRIS buffer.
Dependencies are inferred from the claim text.