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Company intelligence / Profile preview
Orphan Disease Treatment Institute
Tokyo, Tokyo, Japan
- Ownership
- Private
- Development stage
- Other
01
Overview
The company is developing antisense oligonucleotide therapies for Duchenne muscular dystrophy, with its lead candidate DS-5141b/renadirsen having completed or being in Phase 1/2 clinical trials. The focus is on exon skipping approaches targeting the DMD gene to restore functional dystrophin protein expression.
- Therapeutic areas
- Rare Diseases
- Modalities
- Antisense oligonucleotideSmall molecule
- Industry
- Biotech
02
Partnerships
Daiichi SankyoInnovation Network Corporation of JapanMitsubishi UFJ Capital Co., Ltd.
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