US 2026/0218236 A1Published application

Compositions and methods for the treatment of metabolic liver disorders

AssigneePoseida Therapeutics, Inc.

InventorsJingjing JIANG, Julian David DOWN, Xianghong LI, Eric M. OSTERTAG

Life of this patent

Estimating the term…

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).

Claims21

Dependent claims

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

(i)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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:
126;
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:
129;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4;
(ii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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 poly A 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:
2;
or
iii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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:
13;
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:
131;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4.

64 more elements

A method of treating at least one MLD in a subject in need thereof, the method comprising administering to the subject:

a) an adeno-associated virus (AAV) piggyBac transposon polynucleotide comprising in the 5′ to 3′ direction:
(i)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
b) a first piggy Bac 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:
126;
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:
129;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4;
(ii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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 poly A 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:
2;
or
iii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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:
13;
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:
131;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4;
and
b) an AAV transposase polynucleotide comprising in the 5′ to 3′ direction:
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
127;
b) at least one promoter sequence at least one promoter sequence comprising the nucleic acid sequence of SEQ ID NO:
126;
c) at least one transposase sequence comprising the nucleic acid sequence of SEQ ID NO:
48;
d) a polyA sequence comprising the nucleic acid sequences of SEQ ID NO:
136;
e) at least one DNA spacer sequence comprising the nucleic acid sequences of SEQ ID NO:
137;
and
f) a second AAV ITR sequence comprising the nucleic acid sequences of SEQ ID NO:
4.

80 more elements

Dependencies are inferred from the claim text.

Facts

Application: 19/434,421Filed 29 Dec 2025
Priority: 4 Mar 2020
Published: 30 Jul 2026
Term ends: Not estimatedThe source records don’t support an estimate.

Classification19 codes

C12N 15/86
For animal cells: viral vectors
A61K 48/0058
Characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered: nucleic acids adapted for tissue…
A61P 1/16
Drugs for disorders of the alimentary tract or the digestive system: for liver or gallbladder disorders, e.g. hepatoprotective agents,…
C12N 9/1018
Transferring one-carbon groups: carboxy- and carbamoyl transferases

Family

Application62/985,047Us provisional application · filed 4 Mar 2020
Application63/121,488Us provisional application · filed 4 Dec 2020
Application17/909,101Continuation · filed 4 Mar 2021
Application19/434,421Continuation
US 12,540,335 B2Related patent
US 2023/0104091 A1Published application
US 2026/0218236 A1This patent

From the US filing history. Foreign family members aren’t listed.

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Abstract

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

(i)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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:
126;
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:
129;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4;
(ii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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 poly A 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:
2;
or
iii)
a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
3;
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:
13;
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:
131;
and
j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
4.

64 more elements

Facts

Application

19/434,421Filed 29 Dec 2025

Priority

4 Mar 2020

Published

30 Jul 2026

Term ends

Not estimatedThe source records don’t support an estimate.

Classification19 codes

C12N 15/86
For animal cells: viral vectors
A61K 48/0058
Characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered: nucleic acids adapted for tissue…
A61P 1/16
Drugs for disorders of the alimentary tract or the digestive system: for liver or gallbladder disorders, e.g. hepatoprotective agents,…
C12N 9/1018
Transferring one-carbon groups: carboxy- and carbamoyl transferases

Family

Application62/985,047Us provisional application · filed 4 Mar 2020

Application63/121,488Us provisional application · filed 4 Dec 2020

Application17/909,101Continuation · filed 4 Mar 2021

Application19/434,421Continuation

US 12,540,335 B2Related patent

US 2023/0104091 A1Published application

US 2026/0218236 A1This patent

From the US filing history. Foreign family members aren’t listed.