Compositions and methods for treating mitochondrial neurogastrointestinal encephalopathy
AssigneeEntrada Therapeutics, Inc.
InventorsNatarajan SETHURAMAN, Jason RUTH, Lou A. TARTAGLIA, Dehua PEI, Ziqing QIAN
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Abstract
Disclosed herein are compositions and methods of treating disclosure provides for compounds for use in treating Mitochondrial Neurogastrointestinal Encephalopathy Syndrome (MNGIE). In some embodiments, the compounds have cell penetrating activity and thymidine phosphorylase activity. In certain embodiments, the compounds disclosed herein comprise: a) at least one cell-penetrating peptide (CPP) moiety; and b) a thymidine phosphorylase, or an active fragment or analog thereof (TP), wherein the CPP is coupled, directly or indirectly, to TP.
Claims20
A compound having a structure according to Formula I-A:
- wherein:
- CPP is a cell-penetrating peptide;
- TP is thymidine phosphorylase, or an active fragment or analog thereof, and
- L is a linker;
- and
- wherein L is a covalently bound to the side chain of an amino acid on the CPP and to the N-terminus of the TP.
Dependencies are inferred from the claim text.