IP Library › Granted Patent US 12,540,335
Granted Patent B2
US 12,540,335 · App. 17/909,101 · Granted Feb 3, 2026

Compositions and methods for the treatment of metabolic liver disorders

Inventors: Jingjing Jiang (San Diego, CA); Julian Down (San Diego, CA); Xianghong Li (San Diego, CA); Eric M. Ostertag (San Diego, CA)
Assignee: Poseida Therapeutics, Inc.
C12N15/86A61K48/0058A61P1/16C12N9/1018C12N9/1241C12Y201/03003
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Quick Facts
Patent No.
US 12,540,335
App. No.
17/909,101
Granted
Feb 3, 2026
Kind
B2
Abstract

The present disclosure is directed to compositions and methods for the treatment of Metabolic Liver Disorders. The compositions and methods can comprise an adeno-associated virus (AAV) piggyBac polynucleotide comprising a transgene. The transgene may comprise ornithine transcarbamylase (OTC) or methylmalonyl-CoA mutase (MUT1).

Claims (28)

1 . An adeno-associated virus (AAV) piggyBac transposon polynucleotide comprising in the 5′ to 3′ direction:

a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO: 93;

b) a first piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO: 125;

c) a first insulator sequence comprising the nucleic acid sequence of SEQ ID NO: 7;

d) at least one promoter sequence comprising the nucleic acid sequence of SEQ ID NO: 132;

e) at least one transgene sequence comprising the nucleic acid sequence of SEQ ID NO: 22;

f) a polyA sequence comprising the nucleic acid sequence of SEQ ID NO: 97;

g) a second insulator sequence comprising the nucleic acid sequence of SEQ ID NO: 8;

h) a second piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO: 96;

i) at least one DNA spacer sequence comprising the nucleic acid sequence of SEQ ID NO: 130; and

j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO: 94.

2 . The AAV piggyBac transposon polynucleotide of claim 1 , wherein the first piggyBac ITR sequence comprises the nucleic acid sequence of SEQ ID NO: 95.

3 . The AAV piggyBac transposon polynucleotide of claim 1 , wherein the AAV piggyBac transposon polynucleotide comprises nucleotide residues 12 to 4782 of SEQ ID NO: 139.

4 . The AAV piggyBac transposon polynucleotide of claim 3 , wherein the AAV piggyBac transposon polynucleotide comprises of the nucleic acid sequence put forth in SEQ ID NO: 139.

5 . A vector comprising the AAV piggyBac transposon polynucleotide of claim 1 .

6 . The vector of claim 5 , wherein the vector is a viral vector, wherein the viral vector is an AAV viral vector.

7 . The vector of claim 6 , wherein the AAV viral vector is an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10 or AAV11 viral vector.

8 . The vector of claim 6 , wherein the AAV viral vector is an AAV-KP-1 or AAV-NP59 viral vector.

9 . The vector of claim 6 , wherein the AAV viral vector is an AAV-KP-1 viral vector.

10 . The vector of claim 6 , wherein the AAV viral vector is an AAV8 viral vector.

11 . The vector of claim 6 , wherein the AAV viral vector is an AAV9 viral vector.

12 . A composition comprising the vector of claim 5 .

13 . A method of treating Ornithine Transcarbamylase (OTC) Deficiency in a subject in need thereof comprising administering to the subject at least one therapeutically effective dose of the polynucleotide of claim 1 .

14 . The method of claim 13 , wherein the AAV piggyBac transposon polynucleotide comprises nucleotide residues 12 to 4782 of SEQ ID NO: 139.

15 . The method of claim 14 , wherein the AAV piggyBac transposon polynucleotide comprises the nucleic acid sequence put forth in SEQ ID NO: 139.

16 . A method of treating Ornithine Transcarbamylase (OTC) Deficiency in a subject in need thereof comprising administering to the subject at least one therapeutically effective dose of the vector of claim 6 .

17 . The method of claim 16 , wherein the AAV piggyBac transposon polynucleotide comprises nucleotide residues 12 to 4782 of SEQ ID NO: 139.

18 . The method of claim 17 , wherein the AAV piggyBac transposon polynucleotide comprises the nucleic acid sequence put forth in SEQ ID NO: 139.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 25, 2023
From: JIANG, JINGJING; DOWN, JULIAN; LI, XIANGHONG; OSTERTAG, ERIC M.
To: POSEIDA THERAPEUTICS, INC.
Reel/Frame 063761/0475 →
Continuity (3)
Provisional Application 63121488 · Dec 4, 2020
Provisional Application 62985047 · Mar 4, 2020
Related Publication 20230104091A1 · Apr 6, 2023
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