COMPANY RESEARCHRNA-targeted drug developer. Added ION717 and obudanersen targets, diseases and company-reported study phases; these remain investigational.
Profile review: 2026-11-04 · 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.
Ionis describes a portfolio of RNA- and DNA-targeted medicines, with separate pipeline, medicines, clinical-trial, and scientific-publication resources. Product-specific mechanisms and development status should be checked against dated disclosures.
| Program / service | Target / indication | Representative stage | Evidence & scope |
|---|---|---|---|
| ION717 | PRNP · Prion disease | I/IIa期；公司资料 | Human/approved evidence; see original source for scope |
| obudanersen / ION582 | UBE3A-ATS · Angelman syndrome | Phase 3REVEAL；公司资料 | Human/approved evidence; see original source for scope |
Company-name matches are discovery leads; verify entity identity and transaction roles in the original source.
No cataloged update explicitly mentions this company.
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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