Invention Grant
- Patent Title: Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy
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Application No.: US16746403Application Date: 2020-01-17
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Publication No.: US11352626B2Publication Date: 2022-06-07
- Inventor: Elisabeth Marlene Haisma , Marko Potman , Wouter Beumer , Vera Brinks
- Applicant: WINGS THERAPEUTICS, INC.
- Applicant Address: US CA Berkeley
- Assignee: WINGS THERAPEUTICS, INC.
- Current Assignee: WINGS THERAPEUTICS, INC.
- Current Assignee Address: US CA Berkeley
- Agency: Morse, Barnes-Brown & Pendleton, P.C.
- Agent Lisa M. Warren, Esq.; Erin E. Bryan, Esq.
- Priority: GB1504124 20150311
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C07H21/02 ; C12N15/113

Abstract:
Antisense oligonucleotides capable of preventing or reducing exon 73 inclusion into the human COL7A mRNA are characterized in various ways: (a) the oligonucleotide's sequence includes at most two CpG sequences; (b) the oligonucleotide has a length of no more than 24 nucleotides; (c) the oligonucleotide is capable of annealing to the (SRp40/SC35 binding/ESE) element in exon73. These oligonucleotides can usefully be oligoribonucleotides with modified internucleosidic linkages e.g. phosphorothioate linkages.
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