TRANSCRIPTIONAL THERAPY BASED-LIPID NANOPARTICLES AND MRNA FOR THE TREATMENT OF END-STAGE LIVER DISEASE

WIPO Patent Application WO/2023/133489

A8

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.

WO/2003/070750	RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS C VIRUS
WO/2007/140000	ADMINISTRATION OF THE REG1 ANTICOAGULATION SYSTEM
WO/2021/162211	STABILIZER FOR ADENO-ASSOCIATED VIRUSES AND METHOD FOR STABILIZING ADENO-ASSOCIATED VIRUSES BY USING SAME

SOTO-GUTIERREZ ALEJANDRO (US)
BELL AARON W (US)
OSTROWSKA ALINA KRYSTYNA (US)
TAFALENG EDGAR NAOE (US)
FOX IRA JACOB (US)
FACCIOLI LAUNZA ALABY PINHEIRO (US)
HAEP NILS (US)
DIAZ-ARAGON RICARDO (US)
FLORENTINO RODRIGO MACHADO (US)
MOTOMURA TAKASHI (US)
WEISSMAN DREW (US)
ALAMEH MOHAMAD-GABRIEL (US)

PCT/US2023/060211

August 03, 2023

January 06, 2023

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UNIV PITTSBURGH COMMONWEALTH SYS HIGHER EDUCATION (US)
UNIV PENNSYLVANIA (US)

A61K48/00; A61K9/51; A61K31/7115; A61K47/69; A61P1/16; C07K14/05; C12N15/85

SCOTT, Gregory K. et al. (US)

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