PIKFYVE antisense oligonucleotides
AssigneeAcurastem Incorporated
InventorsWen-Hsuan Chang, Emily Elizabeth Lee
Life of this patent
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Abstract
The present invention relates to PIKFYVE antisense oligonucleotides (ASOs), pharmaceutical compositions containing them, and methods for treating, inhibiting, suppressing, and preventing neurological diseases with them.
Claims39
A single stranded antisense oligonucleotide of 20 nucleotides in length that suppresses the expression of a PIKFYVE, wherein the antisense oligonucleotide comprises a nucleobase sequence that comprises the sequence set forth in SEQ ID NO:
- 20.
A single stranded antisense oligonucleotide of 20 nucleotides in length that suppresses the expression of a PIKFYVE, wherein the antisense oligonucleotide has a nucleobase sequence that comprises the sequence set forth in SEQ ID NO:
- 20, wherein the first 3-5 nucleosides at the 5′ end (“5′ wing segment”) comprise modified sugars, the last 3-5 nucleosides at the 3′ end (“3′ wing segment”) comprise modified sugars, and the remaining nucleosides comprise a gap segment.
A single stranded antisense oligonucleotide of 20 nucleotides in length that suppresses the expression of a PIKFYVE, wherein the antisense oligonucleotide comprises the sequence set forth in SEQ ID NO:
- 520, wherein the backbone linkage of the antisense oligonucleotide comprises a mixture of phosphorothioate and phosphodiester linkages.
Dependencies are inferred from the claim text.