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US 11,352,645 B2Granted
Compositions and methods for treating Leber's hereditary optic neuropathy
AssigneeWuhan Neurophth Biotechnology Limited Company
InventorBin Li
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Abstract
Disclosed herein is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein said mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein; and a 3′UTR nucleic acid sequence. Also disclosed is a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.
Claims27
A method of treating an eye disorder, comprising
- a) administering intravitreally or intraocularly to a patient in need thereof a first pharmaceutical composition comprising a therapeutically effective amount of an adeno-associated virus (AAV) comprising a promoter operably linked to a recombinant nucleic acid comprising:
- i) a nucleic acid sequence encoding a mitochondrial targeting peptide;
- ii) a nucleic acid sequence encoding a mitochondrial protein comprising a nucleic acid sequence that is:
- 1) 100% identical to SEQ ID NO:
- 6;
- 2) at least 99% identical to SEQ ID NO:
- 9;
- or
- 3) at least 99% identical to SEQ ID NO:
- 11;
- iii) a 3′UTR nucleic acid sequence;
- and
- b) administering to the patient a second pharmaceutical composition comprising a steroid.
10 more elements
A method of treating Leber's hereditary optic neuropathy (LHON), comprising:
- (a) administering intravitreally or intraocularly to a patient in need thereof a first pharmaceutical composition comprising a therapeutically effective amount of an adeno-associated virus (AAV) comprising a promoter operably linked to a recombinant nucleic acid comprising:
- i) a nucleic acid sequence encoding a mitochondrial targeting peptide, wherein the nucleic acid sequence is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 1-5, or wherein the mitochondrial targeting peptide comprises an amino sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 126-159;
- ii) a nucleic acid sequence encoding a mitochondrial protein comprising a nucleic acid sequence that is:
- 1) 100% identical to SEQ ID NO:
- 6;
- 2) at least 99% identical to SEQ ID NO:
- 9;
- or
- 3) at least 99% identical to SEQ ID NO:
- 11;
- and
- iii) a 3′UTR nucleic acid sequence that is at least 90%, at least 95%, at least 97%, at least 99%, or 100% identical to a sequence selected from the group consisting of SEQ ID NO:
- 13, 14, and 111-125;
- and
- (b) administering to the patient a second pharmaceutical composition comprising methylprednisolone or prednisone.
14 more elements
Dependencies are inferred from the claim text.