Compositions and methods for the treatment of disorders related to cdkl5 deficiency
AssigneeVoyager Therapeutics, Inc.
InventorsMathieu Emmanuel NONNENMACHER, Tyler Christopher MOYER, Jiangyu LI, Dan Richard LAKS
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Abstract
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of CDKL5 proteins via delivery using an adeno-associated virus (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having CDD, or another CDKL5-related disorder.
Claims94
An adeno-associated virus (AAV) particle comprising:
- a) an AAV capsid variant comprising an amino acid sequence having the following formula:
- [N1]-[N2]-[N3], wherein:
- (i) optionally [N1] comprises X1, X2, and X3, wherein at least one of X1, X2, or X3 is G;
- (ii) [N2] comprises the amino acid sequence of SPH;
- and
- (iii) [N3] comprises X4, X5, and X6, wherein at least one of X4, X5, or X6 is a basic amino acid;
- and
- b) a viral genome comprising a cyclin-dependent kinase-like 5 (CDKL5)-encoding sequence.
An adeno-associated virus (AAV) particle comprising a viral genome comprising a cyclin-dependent kinase-like 5 (CDKL5)-encoding sequence and an AAV9 capsid variant comprising the amino acid sequence of SPHSKA (SEQ ID NO:
- 941).
An adeno-associated virus (AAV) particle comprising a viral genome comprising a cyclin-dependent kinase-like 5 (CDKL5)-encoding sequence and an AAV capsid variant comprising:
- (i) a VP1 protein comprising the amino acid sequence of SEQ ID NO:
- 4;
- (ii) a VP2 protein comprising the amino acid sequence of positions 138-742 of SEQ ID NO:
- 4;
- and/or
- (iii) a VP3 protein comprising the amino acid sequence of positions 203-742 of SEQ ID NO:
- 4.
An adeno-associated virus (AAV) particle comprising a viral genome comprising a cyclin-dependent kinase-like 5 (CDKL5)-encoding sequence and an AAV capsid variant comprising:
- (i) a VP1 protein comprising the amino acid sequence of SEQ ID NO:
- 36;
- (ii) a VP2 protein comprising the amino acid sequence of positions 138-742 of SEQ ID NO:
- 36;
- and/or
- (iii) a VP3 protein comprising the amino acid sequence of positions 203-742 of SEQ ID NO:
- 36.
Dependencies are inferred from the claim text.