US 2026/0250351 A1 · TRANSCRIPTIONAL THERAPY BASED-LIPID NANOPARTICLES AND MRNA FOR THE TREATMENT OF END-STAGE LIVER DISEASE

Transcriptional therapy based-lipid nanoparticles and mrna for the treatment of end-stage liver disease

AssigneesUniversity of Pittsburgh—Of the Commonwealth System of Higher Education, The Trustees of the University of Pennsylvania

InventorsAlejandro Soto-Gutierrez, Aaron W. Bell, Alina Krystyna Ostrowska, Edgar Naoe Tafaleng, Ira Jacob Fox, Lanuza Alaby Pinheiro Faccioli, Nils Haep, Ricardo Diaz-Aragon, Rodrigo Machado Florentino, Takashi Motomura, Drew Weissman, Mohamad-Gabriel Alameh

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Abstract

Methods are disclosed for treating end-stage liver failure in a subject that include administering to the subject a therapeutically effective amount of a recombinant mRNA encoding HNF4α isoform 2. The methods can include administering a lipid nanoparticle including the recombinant mRNA to the subject. Also disclosed are recombinant mRNA encoding an HNF4α isoform 1 or HNF4α isoform 2, and lipid nanoparticles include the recombinant mRNA. Methods are also for treating liver disease or end-stage liver failure in a subject that include administering to the subject a therapeutically effective amount of a recombinant mRNA encoding HNF4α isoform 1.

Claims31

Dependent claims

A recombinant mRNA encoding an HNF4α1 or HNF4α2, wherein the recombinant mRNA comprises:

i) a nucleic acid sequence at least 95% identical to one of
a) SEQ ID NO:
2 [HNF4α2],
b) SEQ ID NO:
22 [HNF4α1];
or
c) SEQ ID NO:
30 [HNF4α1];
and
ii) optionally comprises one or more of:
d) a 5′ capping structure,
e) a promoter,
f) a nucleic acid molecule encoding a signal peptide,
g) a 5′ untranslated region (UTR),
h) a 3′ UTR, and
i) a polyA tail.

13 more elements

Dependencies are inferred from the claim text.

Facts

Application

18/726,767Filed 6 Jan 2023

Priority

6 Jan 2022

Published

27 Aug 2026

Term ends

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Classification16 codes

C07K 14/70567
Receptors; Cell surface antigens; Cell surface determinants: nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
C12N 15/86
For animal cells: viral vectors
A61K 38/1783
Receptors; Cell surface antigens; Cell surface determinants: nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
A61K 48/0033
Wherein the non-active part clearly interacts with the delivered nucleic acid: the non-active part being non-polymeric