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US 2026/0098262 A9Published application
Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
AssigneeEntrada Therapeutics, Inc.
InventorsXiang Li, Ziqing Qian, Mahboubeh Kheirabadi, Mark Wysk, Natarajan Sethuraman, Wenlong Lian, Mahsweta Girgenrath, Nelsa Estrella
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Abstract
Described herein in various embodiments are compositions comprising (a) a cyclic peptide; and (b) an antisense compound, wherein the antisense compound targets exon 44 of the DMD gene in a pre-mRNA sequence.
Claims21
1-58. (canceled)
A compound comprising an Endosomal Escape Vehicle (EEV) conjugated to a therapeutic moiety comprising an antisense compound (AC) that is complementary to a target sequence comprising at least a portion of exon 44 of DMD gene in a pre-mRNA sequence;
- wherein the EEV comprises:
- (a) a cyclic peptide of formula:
- or a protonated form thereof,
- wherein
- R1, R2, and R3 are each independently H or a side chain comprising an aryl or heteroaryl group;
- at least two of R1, R2, and R3 are a side chain of phenylalanine;
- AASC is an amino acid side chain;
- R4 and R7 are independently H or an amino acid side;
- each m is independently an integer from 0-3;
- and
- q is an integer from 1-4;
- (b) an exocyclic peptide (EP) comprising from 2 to 10 amino acid residues, wherein 1 or 2 amino acid residues comprise a side chain of a guanidine group, or a protonated form thereof or 2, 3, or 4 lysine residues;
- (c) a linker comprising:
- (i) a —(OCH2CH2)z— subunit, wherein z′ is an integer from 1 to 23;
- (ii) one or more amino acid residues, such as a residue of glycine, β-alanine, 4-aminobutyric acid, 5-aminopentoic acid or 6-aminohexanoic acid, or combinations thereof;
- or
- (iii) combinations of (i) and (ii).
14 more elements
Dependencies are inferred from the claim text.