Wholly owned Nippon Shinyaku subsidiary developing and commercializing rare-disease medicines, including DMD exon-skipping programs.
Profile review: 2026-11-07 · Fields retain their individual source dates. Undisclosed values are not zero; early or historical events do not establish the latest financing. Awards and acquisitions are distinguished from equity rounds.
Disease tags match recorded text mentions and require program-level verification.
VILTEPSO received US accelerated approval based on increased muscle dystrophin; continued approval may depend on confirmatory clinical-benefit evidence.
| Program / service | Target / indication | Representative stage | Evidence & scope |
|---|---|---|---|
| VILTEPSO / viltolarsen | DMD exon 53 · DMD amenable to exon 53 skipping | 美国加速批准；2020-08-12 | See original evidence scope |
| NS-089 / NCNP-02 (brogidirsen) | DMD exon 44 · DMD | Phase 2；公司管线页 | See original evidence scope |
| NS-050 / NCNP-03 | DMD exon 50 · DMD | Phase 1/2；公司管线页 | See original evidence scope |
| NS-051 / NCNP-04 | DMD exon 51 · DMD | Preclinical；公司管线页 | See original evidence scope |
Company-name matches are discovery leads; verify entity identity and transaction roles in the original source.
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Phio announced a November 7 SITC oral presentation of final Phase 1b findings for intratumoral PH-762, its PD-1-silencing siRNA candidate, in cutaneous carcinomas. The company described tolerability and potential tissue-sparing neoadjuvant use, while stating that additional safety and efficacy studies are needed. A Phase 2b study is planned for the first half of 2027.
Vir Biotechnology announced that Phase 3 ECLIPSE 1 results for elebsiran plus tobevibart in chronic hepatitis delta will be presented on November 8, with an investor discussion on November 9. Elebsiran is an HBV-targeting siRNA; tobevibart is an antibody. The release announces the forthcoming readout and expanded Phase 2 presentations but does not disclose the Phase 3 numerical results.
A Nature Communications study reports a peptide–oligonucleotide nanohybrid for bone-targeted miR-26a-5p delivery. The original abstract describes reduced GSK-3β expression and stabilization of HIF-1α, supporting coupled bone formation and angiogenesis. Systemic administration limited skeletal and vascular injury in a rat glucocorticoid-induced bone-loss model. This is preclinical miRNA delivery, not oral cyclic peptide therapy or human efficacy evidence.
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