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US 2026/0091139 A1Published application
Treatment of cardiomyopathy with aav gene therapy vectors
AssigneeDiNAQOR AG
InventorsPeter Cameron COLOSI, Pooja AGARWAL, Wesley YONEMOTO, Sergio VIEDMA-GONZALEZ, Eduard AYUSO, Thomas VOIT, Valeria RICOTTI
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Abstract
Provided herein are gene therapy compositions and methods of treating reduced levels of functional cardiac myosin binding protein C in a subject having hypertrophic cardiomyopathy.
Claims27
A recombinant vector construct comprising:
- a nucleic acid encoding a functional human cardiac myosin binding protein C (cMyBP-C) comprising an amino acid sequence at least 95% identical to SEQ ID NO:
- 2, or complement thereof, operably linked to:
- a heterologous cardiomyocyte-specific transcription regulatory region comprising a fragment or variant of a hTNNT2 promoter,
- a polyadenylation signal, and
- one or both of 5′ and 3′ AAV inverted terminal repeat (ITR) sequences.
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A method of producing an rAAV particle comprising the steps of:
- (a) providing a cell permissive for AAV replication with one or more nucleic acid constructs comprising:
- (i) a recombinant vector construct comprising (1) at least one AAV ITR, (2) a heterologous cardiomyocyte-specific transcription regulatory region, and (3) a nucleic acid encoding a functional human cardiac myosin binding protein C,
- (ii) a nucleotide sequence encoding one or more AAV Rep proteins which is operably linked to a promoter that is capable of driving expression of the Rep protein(s) in the cell;
- and
- (iii) a nucleotide sequence encoding one or more AAV capsid proteins which is operably linked to a promoter that is capable of driving expression of the capsid protein(s) in the cell;
- (b) culturing the cell under conditions permitting expression of the Rep and the capsid proteins;
- and
- (c) recovering the rAAV particle.
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Dependencies are inferred from the claim text.