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US 2022/0090129 A1Published application
Recombinant adeno-associated viral vector for gene delivery
AssigneeAbeona Therapeutics Inc.
InventorsTimothy J. MILLER, Linas PADEGIMAS
Life of this patent
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Abstract
Provided herein are recombinant AAV vectors, AAV viral vectors, and capsid proteins for improved gene therapy, and methods for their manufacture and use.
Claims28
A nucleic acid encoding an AAV capsid protein comprising a VP1 portion, a VP2 portion and a VP3 portion, wherein the VP3 portion comprises variable regions (VR) I to IX wherein:
- (SEQ ID NO:
- 54)
- (a) VR-II comprises amino acid sequence DNNGVK,
- (SEQ ID NO:
- 55)
- (b) VR-III comprises amino acid sequence NDGS,
- (SEQ ID NO:
- 56)
- (c) VR-IV comprises amino acid sequence
- INGSGQNQQT,
- (SEQ ID NO:
- 57)
- (d) VR-V comprises amino acid sequence
- RVSTTTGQNNSNFAWTA,
- (SEQ ID NO:
- 58)
- (e)VR-VI comprises amino acid sequence
- HKEGEDRFFPLSG,
- (SEQ ID NO:
- 59)
- (f) VR-VII comprises amino acid sequence
- KQNAARDNADYSDV,
- (SEQ ID NO:
- 60)
- (g) VR-VIII comprises amino acid sequence
- ADNLQQQNTAPQI, and
- (SEQ ID NO:
- 61)
- (h) VR-IX comprises amino acid sequence
- NYYKSTSVDF.
27 more elements
An AAV viral vector comprising
- (i) an AAV capsid protein having the amino acid sequence of SEQ ID NO:2, SEQ ID NO:
- 3, SEQ ID NO:
- 31, SEQ ID NO:
- 32, SEQ ID NO:
- 33, or SEQ ID NO:
- 34 and
- (ii) an AAV vector, wherein the AAV vector comprises in 5′ to 3′ orientation,
- (a) a first AAV inverted terminal repeat,
- (b) a promoter,
- (c) a heterologous nucleic acid,
- (d) a poly-A tail;
- and
- (e) a second AAV inverted terminal repeat.
10 more elements
Dependencies are inferred from the claim text.