Compositions and methods for treating stxbp1 disorders
AssigneesBiomarin Pharmaceutical Inc., Deep Genomics Incorporated
InventorsJan Hsi LUI, Jonathan LEBOWITZ, Guoying Karen YU, Xing TANG, Arthur WUSTER, Yumaine CHONG, Erle Marie HOLGERSEN, Mark George Ford SUN, Janine Jia-Ying TRUONG
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Abstract
Provided herein are antisense oligonucleotides (ASOs) and pharmaceutically acceptable derivatives thereof that modulate expression of STXBP1 and have activity in treating STXBP1 disorders. Also provided are pharmaceutical compositions containing the ASOs and methods of using the ASOs for treating a subject with an STXBP1 disorder.
Claims24
An antisense oligonucleotide (ASO) comprising a base sequence that is (i) complementary or reverse complementary to a portion of STXBP1 mRNA or pre-mRNA, or (ii) at least 90% complementary or reverse complementary to the portion of STXBP1 mRNA or pre-mRNA, or (iii) has 1 or 2 mismatches with the portion of STXBP1 mRNA or pre-mRNA or complement thereof.
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Dependencies are inferred from the claim text.