US2023270685A1PendingUtilityA1

POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE

82
Assignee: MODERNATX INCPriority: Dec 17, 2015Filed: Nov 18, 2022Published: Aug 31, 2023
Est. expiryDec 17, 2035(~9.4 yrs left)· nominal 20-yr term from priority
Inventors:Paolo MartiniVladimir PresnyakKerry BenenatoStephen HogeIain McfadyenEllalahewage Sathyajith Kumarasinghe
A61K 48/005C12N 15/88A61K 9/5123A61K 9/1272A61K 38/52A61P 3/00A61P 43/00C12N 9/90C12N 15/52C12Y 504/99002A61K 38/00
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Claims

Abstract

The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.

Claims

exact text as granted — not AI-modified
1 - 15 . (canceled) 
     
     
         16 . A method of treating methylmalonic acidemia in a human subject in need thereof, the method comprising administering to the human subject once every week a therapeutically effective amount of a lipid nanoparticle comprising a messenger RNA (mRNA) encoding the human methylmalonyl-CoA mutase (MCM) polypeptide of SEQ ID NO:208, SEQ ID NO:209, SEQ ID NO:210, SEQ ID NO: 211, SEQ ID NO:212, or SEQ ID NO:213. 
     
     
         17 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:208. 
     
     
         18 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:209. 
     
     
         19 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:210. 
     
     
         20 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:211. 
     
     
         21 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:212. 
     
     
         22 . The method of  claim 16 , wherein the mRNA encodes the human MCM polypeptide of SEQ ID NO:213. 
     
     
         23 . The method of  claim 16 , wherein the mRNA comprises at least one chemically modified nucleobase. 
     
     
         24 . The method of  claim 23 , wherein the at least one chemically modified nucleobase comprises N1-methylpseudouracil. 
     
     
         25 . The method of  claim 23 , wherein the at least one chemically modified nucleobase comprises 5-methoxyuracil.

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