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Orphan Disease Treatment Institute

Tokyo, Tokyo, Japanorphandiseasecenter.med.upenn.edu ↗

Ownership: Private
Development stage: Other

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

Partnerships

Daiichi SankyoInnovation Network Corporation of JapanMitsubishi UFJ Capital Co., Ltd.

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