Modified polynucleotides for treating lipase a, lysosomal acid, cholesterol esterase protein deficiency
AssigneeModerna Therapeutics, Inc.
InventorsStephane Bancel, Tirtha Chakraborty, Antonin de Fougerolles, Susan Whoriskey
Life of this patent
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Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Claims9
A method of producing a polypeptide of interest in a mammalian cell or tissue comprising contacting said mammalian cell or tissue with a modified mRNA, wherein said modified mRNA encodes a member selected from the group consisting of lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1127), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1128), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1129), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1130), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1131), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1132), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1133), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1134), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1135), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1136) and lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1137), and wherein said modified mRNA comprises modifications selected from the group consisting of:
- (a) 5-methylcytosine and 2-thiouridine,
- (b) pseudouridine and 5-methylcytosine, and
- (c) 1-methylpseudouridine.
A modified mRNA encoding a polypeptide of interest, said polypeptide of interest selected from the group consisting of lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1127), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1128), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1129), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1130), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1131), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1132), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1133), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1134), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1135), lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1136) and lipase A, lysosomal acid, cholesterol esterase (SEQ ID NO:
- 1137), and wherein said modified mRNA comprises modifications selected from the group consisting of:
- (a) 5-methylcytosine and 2-thiouridine,
- (b) pseudouridine and 5-methylcytosine, and
- (c) 1-methylpseudouridine.
(canceled)
Dependencies are inferred from the claim text.