Exon skipping oligomer conjugates for muscular dystrophy
NZ795532BActive Publication Date: 2026-09-29SAREPTA THERAPEUTICS INC
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Patent Information
- Application Number
- NZ795532
- Authority / Receiving Office
- NZ · NZ
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2017-09-22
- Filing Date
- 2017-12-13
- Publication Date
- 2026-09-29
- Estimated Expiration
- 2037-12-13
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Figure 1_ABST
Abstract
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described in Formula (I). The antisense oligomer conjugate has addition of an R6-G-peptide group with the terminal acetyl group at the 3' end. In particular, the invention relates to antisense oligomer conjugates of Formula (III).
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