IP Library Granted Patent US 12,553,062
Granted Patent B2
US 12,553,062 · App. 17/258,516 · Granted Feb 17, 2026

AAV compositions

Inventors: Eva Fritscher (Vienna, AT); Christian Fiedler (Vienna, AT); Tanvir Tabish (Vienna, AT); Dominik Mittergradnegger (Vienna, AT); Stefan Reuberger (Vienna, AT)
Assignee: TAKEDA PHARMACEUTICAL COMPANY LIMITED
C12N15/86A61K47/183A61K47/26C12N2750/14143
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Quick Facts
Patent No.
US 12,553,062
App. No.
17/258,516
Granted
Feb 17, 2026
Kind
B2
Abstract

The AAV compositions comprising an AAV and a buffer. These AAV compositions are appropriate for us in intracerebroventricular (ICV) and intraparenchymal (IPA) administration, as well as other routes of administration and can be provided in frozen, liquid or potentially freeze dried (lyophilized) state.

Claims (17)

1 . A storage stable aqueous composition comprising an adeno-associated virus (AAV) vector and a buffer formulation consisting of:

(a) about 30 mM to about 50 mM of L-histidine;

(b) about 100 mM to about 160 mM of sodium chloride;

(c) about 0.0001% (w/w) to about 0.01% (w/w) of polysorbate 80 (PS80); and

(d) water,

wherein the composition does not comprise sugar, sugar alcohol, or a combination thereof,

wherein the composition has a pH of 6.9 to 7.4, and

wherein the AAV vector has a titer that is stable when stored at a temperature range of about −60° C. to about 5° C. for at least 6 months.

2 . The aqueous composition of claim 1 , wherein the composition has a pH of about 7.0.

3 . The aqueous composition of claim 1 , wherein the PS80 is about 0.0005% to about 0.006% w/w.

4 . The aqueous composition of claim 1 , comprising the AAV vector and the buffer formulation consisting of: (a) about 50 mM of L-histidine, (b) about 154 mM of sodium chloride, (c) about 0.003% w/w of PS80, and (d) water.

5 . The aqueous composition of claim 1 , comprising: the AAV vector and the buffer formulation consisting of (a) about 30 mM of L-histidine; (b) about 154 mM of NaCl; (c) about 0.001% w/w to about 0.005% w/w of polysorbate 80 (PS80); and (d) water.

6 . The aqueous composition of claim 1 , wherein the AAV vector is an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, or AAV10 vector.

7 . The aqueous composition of claim 1 , wherein the AAV vector is genetically and/or chemically modified.

8 . A lyophilized storage stable composition, wherein the lyophilized storage stable composition is dehydrated composition of the aqueous composition of claim 1 .

9 . The aqueous composition of claim 1 , wherein the composition has an osmolality of 200 to 320 mOsmol/kg.

10 . The aqueous composition of claim 1 , wherein the AAV vector has a titer that is stable when stored at a temperature range of about −20° C. to about 4° C. for at least 6 months.

Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2021
From: BAXALTA GMBH; BAXALTA INCORPORATED
To: TAKEDA PHARMACEUTICAL COMPANY LIMITED
Reel/Frame 055568/0679 →
CHANGE OF ADDRESS Recorded Jan 28, 2021
From: BAXALTA GMBH
To: BAXALTA GMBH
Reel/Frame 055146/0018 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 22, 2021
From: FRITSCHER, EVA; FIEDLER, CHRISTIAN; TANVIR, TABISH; MITTERGRADNEGGER, DOMINIK; REUBERGER, STEFAN
To: BAXALTA INCORPORATED; BAXALTA GMBH
Reel/Frame 054995/0617 →
Continuity (2)
Provisional Application 62696792 · Jul 11, 2018
Related Publication 20210163990A1 · Jun 3, 2021
References Cited (50)
US 6764845B2 · Sista · 2004 [cited by applicant]
US 9051542B2 · Wright · 2015 [cited by applicant]
US 20020041884A1 · Evans · 2002 [cited by examiner]
US 20030153065A1 · Kovesdi et al. · 2003 [cited by applicant]
US 20110076744A1 · Wright · 2011 [cited by examiner]
US 20110229455A1 · Matthiessen · 2011 [cited by examiner]
US 20120141528A1 · Coffey · 2012 [cited by applicant]
US 20150224155A1 · Coffey · 2015 [cited by examiner]
US 20160199496A1 · Jezek et al. · 2016 [cited by applicant]
US 20170130208A1 · Potter et al. · 2017 [cited by applicant]
US 20210123028A1 · Knop · 2021 [cited by examiner]
JP 2003525909 · 2003 [cited by applicant]
JP 2011517955 · 2011 [cited by applicant]
JP 2012143233 · 2012 [cited by applicant]
JP 2014501493 · 2014 [cited by applicant]
WO 1989006542 · 1989 [cited by applicant]
WO 199510601 · 1995 [cited by applicant]
WO 199945966 · 1999 [cited by applicant]
WO 200166137 · 2001 [cited by applicant]
WO 2001066137 · 2001 [cited by applicant]
WO 2005118792 · 2005 [cited by applicant]
WO 2013036961 · 2013 [cited by applicant]
WO 2013130393 · 2013 [cited by applicant]
WO 2015040234 · 2015 [cited by applicant]
WO WO2015040234A1 · 2015 [cited by examiner]
WO 2015121501 · 2015 [cited by applicant]
WO 2016154055 · 2016 [cited by applicant]
WO WO2017096039A1 · 2017 [cited by examiner]
WO 20180128689A1 · 2018 [cited by applicant]
WO WO2018128689A1 · 2018 [cited by examiner]
Sigma catalogue (www.sigmaaldrich.com/US/en/support/ calculators-and-apps/1x-phosphate-buffered-saline, pp. 1-2 (Year: 2017). [cited by examiner]
Evans et al (Journal of Pharmaceutical Science, 93(10), 2458-2475 (Year: 2004). [cited by examiner]
First Office Action issued Jun. 20, 2023 in connection with Japanese Application No. 2020-572381. [cited by applicant]
First Office Action issued Jul. 3, 2023 in connection with Japanese Application No. 2022-113804. [cited by applicant]
International Search Report mailed Sep. 27, 2019 in connection with PCT/US19/041398. [cited by applicant]
Written Opinion mailed Sep. 27, 2019 in connection with PCT/US19/041398. [cited by applicant]
Office Action issued Jun. 13, 2023 in connection with. Russian Patent Application No. 2020142674. [cited by applicant]
Supplementary Search Report mailed May 9, 2022 in connection with EP 19833959.0. [cited by applicant]
Decision of Rejection issued Apr. 14, 2022 in connection with JP Application No. 2019-522442. [cited by applicant]
Wright J.F. et al., “Identification of factors that contribute to recombinant AAV2 particle aggregation and tmethods to prevent its occurrence during vectoor purificcation and formulation”, Molecular Therapy: The Journa… [cited by applicant]
International Preliminary Report on Patentability mailed May 7, 2019 in connection with International Application No. PCT/US17/59971. [cited by applicant]
International Search Report and Written Opinion mailed Jun. 28, 2018 in connection with PCT/US17/059971. [cited by applicant]
Monahan, Gene therapy in an era of emerging treatment options for hemophilia B, J. Thromb. Haemost, Jun. 2015: 13 ; S151-S160. [cited by applicant]
Rayaprolu et al., Comparative analysis of adeno-associated virus capsid stability and dynamics, J. Virol. Dec. 2013; 87 (24):13150-160 Epub Sep. 25, 2013. [cited by applicant]
International Preliminary Report on Patentability issued Jan. 12, 2021 in connection with PCT/US19/041398. [cited by applicant]
Office Action issued Jan. 7, 2021 in connection with U.S. Appl. No. 16/347,107. [cited by applicant]
Final Office Action issued Jul. 7, 2021 in connection with U.S. Appl. No. 16/347,107. [cited by applicant]
Office Action and Search Report mailed Jan. 20, 2023 in connection with Russian Patent Application No. 2020142674. [cited by applicant]
Wang L. et al. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl. Acad. Sci. USA, Mar. 30, 1999; 96(7): 3906-3910. doi: 10.1073/pnas.96.7.3906. [cited by applicant]
Office Action issued Nov. 22, 2021 in connection with U.S. Appl. No. 16/347,107. [cited by applicant]