Central nervous system targeting polynucleotides
AssigneeVoyager Therapeutics, Inc.
InventorsGregory Robert Stewart, Dinah Wen-Yee Sah, Jinzhao Hou, Adrian Philip Kells, Martin Goulet, Kartik Ramamoorthi, Pengcheng Zhou, Yanqun Shu
Life of this patent
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Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding payloads for the treatment of CNS disorders.
Claims47
1-2. (canceled)
The polynucleotide of claim 7, wherein the polynucleotide comprises at least one 5′ inverted terminal repeat (ITR), one 3′ inverted terminal repeat ITR, a promoter region, and an enhancer, and wherein said enhancer is a CMV enhancer.
5-6. (canceled)
A polynucleotide encoding human frataxin comprising a sequence having at least 95% identity to a sequence selected from the group consisting of SEQ ID NOs 1-94 or variants thereof.
A polynucleotide comprising a sequence selected from the group consisting of SEQ ID NOs 1-94, wherein the frataxin payload is replaced by gene encoding a CNS protein of interest or a modulatory nucleic acid.
(canceled)
13-18. (canceled)
The method of claim 18, wherein the polynucleotide in packaged in an AAV particle, said AAV particle comprising a capsid having a capsid serotype selected from the group consisting of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hu14), AAV10, AAV11, AAV12, AAVrh8, AAVrh10, AAV-DJ and AAVDJ8.
20-24. (canceled)
The method of claim 26, wherein increasing the level of protein treats a disease or disorder, wherein the disease or disorder is Friedreich's Ataxia.
A method of increasing the level of a protein in the CNS of a subject in need thereof comprising administering to said subject via prolonged infusion an effective amount of an AAV particle comprising a viral genome encoding a sequence selected from the group consisting of SEQ ID NOs 1-94 or a therapeutically effective amount of a polynucleotide comprising a sequence selected from the group consisting of SEQ ID NOs 1-94 wherein the frataxin payload has been replaced by gene encoding a CNS protein of interest or a modulatory nucleic acid.
30-32. (canceled)
35-41. (canceled)
43-48. (canceled)
50-53. (canceled)
(canceled)
60-62. (canceled)
64-67. (canceled)
69-78. (canceled)
(canceled)
82-88. (canceled)
92-93. (canceled)
95-101. (canceled)
Dependencies are inferred from the claim text.