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US 2024/0391966 A1Published application
Materials and methods for treating friedreich's ataxia
AssigneeThe Trustees of Indiana University
InventorRonald Mark Payne
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Abstract
A TAT-FXN fusion polypeptide useful in treating subjects diagnosed with Friedrich's Ataxia, hypertrophic cardiomyopathy, or both are disclosed, as are related methods of treatment and pharmaceutical compositions.
Claims30
A fusion polypeptide, comprising:
- a first peptide having an amino acid sequence with at least 90% sequence identity to the amino acid sequence of SEQ ID NO:
- 2;
- and
- a second peptide having an amino acid sequence with at least 90% sequence identity to the amino acid sequence of SEQ ID NO:
- 4;
- wherein the first peptide is fused through a 2-amino acid linker to the second peptide.
3 more elements
A fusion polypeptide, consisting of:
- the first peptide having an amino acid sequence with at least 90% sequence identity to the amino acid sequence of SEQ ID NO:
- 2;
- the second peptide having an amino acid sequence with at least 90% sequence identity to the amino acid sequence of SEQ ID NO:
- 4;
- and
- a 2 amino acid linker disposed between the first and second peptides.
3 more elements
Dependencies are inferred from the claim text.