US 12,403,164 B2
Lentiviral vector formulations
Andrew Kroetsch, Waltham, MA (US); and Isidro Zarraga, Waltham, MA (US)
Assigned to BIOVERATIV THERAPEUTICS INC., Waltham, MA (US); FONDAZIONE TELETHON, Rome (IT); and OSPEDALE SAN RAFFAELE S.R.L., Milan (IT)
Filed by BIOVERATIV THERAPEUTICS INC., Waltham, MA (US); FONDAZIONE TELETHON, Rome (IT); and OSPEDALE SAN RAFFAELE S.R.L., Milan (IT)
Filed on Sep. 30, 2020, as Appl. No. 17/038,031.
Claims priority of provisional application 62/908,390, filed on Sep. 30, 2019.
Prior Publication US 2021/0113634 A1, Apr. 22, 2021
Int. Cl. A61K 39/205 (2006.01); A61K 35/76 (2015.01); A61K 47/26 (2006.01); A61P 7/04 (2006.01); C12N 15/86 (2006.01)
CPC A61K 35/76 (2013.01) [A61K 47/26 (2013.01); A61P 7/04 (2018.01); C12N 15/86 (2013.01)] 15 Claims
OG exemplary drawing
 
1. 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 VIII (FVIII) coding sequence set forth in SEQ ID NO: 1 or SEQ ID NO: 2;
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.