US 12,377,136 B2 · Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria

Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria

AssigneesModernaTX, Inc., FundacióN Para La InvestigacióN MéDica Aplicada

InventorsPaolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Lei Jiang, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Antonio Fontanellas Roma, Pedro Berraondo Lopez, Matias Antonio Avila Zaragoza, Lin Tung Guey, Staci Sabnis

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Abstract

The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).

Claims11

Dependent claims

A method of treating an acute Porphyria attack in a human subject in need thereof, the method comprising administering to the human subject a lipid nanoparticle comprising a therapeutically effective amount of a messenger RNA (mRNA) encoding a porphobilinogen deaminase (PBGD) polypeptide, wherein the mRNA comprises an open reading frame comprising the nucleotide sequence set forth in SEQ ID NO:

102 or SEQ ID NO:
104, and wherein the human subject has acute intermittent Porphyria.

Dependencies are inferred from the claim text.

Facts

Application

17/813,984Filed 21 Jul 2022 · continuation

Priority

EP 173822599 May 2017

Published

US 2023/0112986 A113 Apr 2023

Granted

5 Aug 2025

Term ends

Not estimatedThe source records don’t support an estimate.

Classification11 codes

A61K 38/45
Enzymes; Proenzymes; Derivatives thereof: transferases
A61K 48/0033
Wherein the non-active part clearly interacts with the delivered nucleic acid: the non-active part being non-polymeric
A61K 48/005
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy:…
A61P 7/08
Drugs for disorders of the blood or the extracellular fluid: plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for…

Family

PCT/US2017/033418Parent of this continuation · filed 18 May 2017

US 2023/0112986 A1Pre-grant publication · published 13 Apr 2023

Application16/302,339Parent of this continuation

US 2020/0085916 A1Published application

US 12,377,136 B2This patent

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