US2019106700A1PendingUtilityA1

siRNA And Their Use In Methods And Compositions For Inhibiting The Expression Of The ORAI1 Gene

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Assignee: SYLENTIS SAUPriority: Oct 22, 2013Filed: Dec 14, 2018Published: Apr 11, 2019
Est. expiryOct 22, 2033(~7.3 yrs left)· nominal 20-yr term from priority
Inventors:Ana I. JiménezCovadonga PañedaTamara Martinez
A61P 37/08C12N 2310/14C12N 2310/335C12N 2310/321C12N 2310/315A61K 31/7105C12N 15/113A61P 27/14C12N 2310/50C12N 15/1138C12N 2310/33A61P 27/02C12N 2320/30C12N 2310/3521
47
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Claims

Abstract

The invention relates to siRNA molecules and their use in methods and pharmaceutical compositions for inhibiting the expression of the ORAil gene. The invention also relates to the use of said siRNAs molecules in the treatment and/or prevention of an eye condition characterised by increased expression and/or activity of ORAil gene, preferably said eye condition is conjunctivitis and/or an ocular allergy such as seasonal allergic conjunctivitis, perennial allergic conjunctivitis, vernal keratoconjunctivitis, atopic keratoconjunctivitis, and giant papillary conjunctivitis.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . An siRNA molecule which targets SEQ ID NO. 1 and reduces the expression of the ORAI1 gene when introduced into a cell. 
     
     
         2 . The siRNA molecule according to  claim 1 , wherein said siRNA comprises a 19 nucleotide double-stranded region. 
     
     
         3 . The siRNA molecule according to  claim 2 , wherein said siRNA is blunt-ended. 
     
     
         4 . The siRNA molecule according to  claim 1 , wherein said siRNA comprises the nucleotide sequence of SEQ ID NO. 112. 
     
     
         5 . The siRNA molecule according to  claim 1 , wherein at least one nucleotide comprises a chemical modification. 
     
     
         6 . The siRNA molecule according to  claim 5 , wherein said chemical modification is 2′-O-methylation; substitution of a uracyl ribose nucleotide with a deoxythymidine nucleotide; or a combination thereof. 
     
     
         7 . The siRNA molecule according to  claim 6 , wherein said chemical modification is on the sense strand, the antisense strand, or on both the sense strand and the antisense strand. 
     
     
         8 . The siRNA molecule according to  claim 3 , wherein said siRNA has a nucleotide sequence that consists of SEQ ID NO. 112. 
     
     
         9 . The siRNA molecule according to  claim 8 , wherein said siRNA has at least one nucleotide comprising a chemical modification. 
     
     
         10 . The siRNA molecule according to  claim 9 , wherein said chemical modification is 2′-O-methylation; substitution of a uracyl ribose nucleotide with a deoxythymidine nucleotide; or a combination thereof. 
     
     
         11 . The siRNA molecule according to  claim 10 , wherein said chemical modification is on the sense strand, the antisense strand, or on both the sense strand and the antisense strand. 
     
     
         12 . The siRNA molecule according to  claim 8 , wherein said siRNA has a nucleotide sequence of one of SEQ ID NO. 223 to SEQ ID NO. 229, SEQ ID NO:233, or SEQ ID NO:235. 
     
     
         13 . A pharmaceutical composition comprising an siRNA molecule which targets SEQ ID NO. 1 and reduces the expression of the ORAI1 gene when introduced into a cell; and a pharmaceutically acceptable carrier. 
     
     
         14 . The pharmaceutical composition of  claim 13 , wherein the siRNA molecule is blunt ended and comprises a 19 nucleotide double-stranded region. 
     
     
         15 . The pharmaceutical composition of  claim 14 , wherein said siRNA has a nucleotide sequence that consists of SEQ ID NO. 112. 
     
     
         16 . The pharmaceutical composition of  claim 15 , wherein said siRNA has at least one nucleotide comprising a chemical modification. 
     
     
         17 . The pharmaceutical composition of  claim 16 , wherein said chemical modification is 2′-O-methylation; substitution of a uracyl ribose nucleotide with a deoxythymidine nucleotide; or a combination thereof. 
     
     
         18 . The pharmaceutical composition of  claim 17 , wherein said chemical modification is on the sense strand, the antisense strand, or on both the sense strand and the antisense strand. 
     
     
         19 . The pharmaceutical composition of  claim 15 , wherein said siRNA has a nucleotide sequence of one of SEQ ID NO. 223 to SEQ ID NO. 229, SEQ ID NO:233, or SEQ ID NO:235. 
     
     
         20 . The pharmaceutical composition of  claim 13  which is formulated for topical administration to the eye. 
     
     
         21 . The pharmaceutical composition of  claim 15  which is formulated for topical administration to the eye.

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