	US 11,939,582 B2
	Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies
Steven Petrou, Eltham (AU)
Assigned to RogCon, Inc., Miami Beach, FL (US)
Appl. No. 17/269,706
Filed by RogCon, Inc., Miami Beach, FL (US)
PCT Filed Aug. 20, 2019, PCT No. PCT/US2019/047313 § 371(c)(1), (2) Date Feb. 19, 2021, PCT Pub. No. WO2020/041348, PCT Pub. Date Feb. 27, 2020.
Claims priority of provisional application 62/765,344, filed on Aug. 20, 2018.
Prior Publication US 2021/0317462 A1, Oct. 14, 2021
Int. Cl. C12N 15/11 (2006.01); A61K 9/00 (2006.01); A61P 25/00 (2006.01); C12N 15/113 (2010.01)

CPC C12N 15/1138 (2013.01) [A61K 9/0085 (2013.01); A61P 25/00 (2018.01); C12N 2310/11 (2013.01); C12N 2310/314 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/341 (2013.01); C12N 2310/346 (2013.01); C12N 2320/32 (2013.01)]

14 Claims

1. A method of treating an SCN1A encephalopathy in a subject in need thereof, the method comprising administering to the subject a composition comprising a single-stranded oligonucleotide, wherein the single-stranded oligonucleotide is 10-80 nucleosides in length and has a nucleobase sequence comprising a portion of 10 contiguous nucleobases having at least 80% complementarity to an equal length portion of a target region of a pre-mRNA transcript or an mRNA transcript of a human SCN2A gene, in an amount and for a duration sufficient to treat the SCN1A encephalopathy, wherein the subject has a loss-of-function mutation in SCN1A, and wherein the single-stranded oligonucleotide is selective for SCN2A pre-mRNA or mRNA over SCN1A pre-mRNA or mRNA.