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US 12,545,929 B2Granted
Lactam-modified adeno-associated virus vectors
AssigneeCoave Therapeutics
InventorsWillem Broekaert, Melanie Glossop, Karl Gibson, Nicolas Ferry, Gaëlle Lefèvre
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Abstract
The present invention relates to adeno-associated virus (AAV) vectors modified by the covalent coupling of at least one compound comprising a lactam moiety (e.g., β-lactam) to at least one amino group of an amino acid residue of the capsid of the AAV vectors. The AAV vectors are useful in transducing a cell, especially for gene therapy.
Claims18
An adeno-associated virus (AAV) vector comprising a moiety according to formula (II):
- or a pharmaceutically acceptable salt thereof, wherein
- N* is a nitrogen atom of an amino group of an amino acid residue of the AAV vector's capsid;
- ----- represents the point of attachment to the AAV vector's capsid;
- Z is a functional moiety comprising a cell-type specific ligand, a labelling agent, a steric shielding agent, a drug moiety or combinations thereof;
- L is a linker;
- R1, R1, R2 and R2′ are each independently hydrogen, halogen, or an optionally substituted C1-6 alkyl;
- or
- R1 and R1′ or R2 and R2′, together with their intervening atoms, may come together of form an optionally substituted spiro-fused ring;
- or
- R1 and R2, together with their intervening atoms, may form an optionally substituted 3- to 7-membered saturated, partially unsaturated, or aryl or heteroaryl ring having 0-2 heteroatoms independently selected from nitrogen, oxygen, or sulfur.
7 more elements
Dependencies are inferred from the claim text.