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8 matching results · plus news and online publication results
Oligonucleotide Therapeutics & Delivery Follow siRNA, ASOs, splice modulation, conjugation, delivery, analytics, and manufacturing. How can sequence, chemistry, and delivery contributions be distinguished? What evidence boundaries matter for extrahepatic delivery? Which analytical questions matter w…
Oligonucleotide Therapeutics & Delivery
…tions Research guide ↗ Oligonucleotide Therapeutics & Delivery Follow siRNA, ASOs, splice modulation, conjugation, delivery, analytics, and manufacturing. Research questions & evidence boundaries Research guide ↗ In Vivo Cell Engineering & Gene Editing Explore in vivo CAR-T, nonviral editing deliver…
Develops gene delivery platforms for mRNA, saRNA, siRNA, and ASOs, along with internal drug programs. In 2026 it announced an LNP platform and Korean-market collaboration with Crystal NAX. Develops gene delivery platforms for mRNA, saRNA, siRNA, and ASOs, along with internal drug programs. In 2026 i…
mRNA / LNP
Develops RNA-activation and siRNA medicines. Representative programs include RAG-01 for NMIBC, RAG-18 for DMD and RAG-17 for SOD1-associated ALS. Stages are tied to dated issuer disclosures; this is not a complete pipeline or independent efficacy validation. Founded in 2017; the company lists Suzhou…
Oligonucleotide Therapeutics & Delivery
…tions Research guide ↗ Oligonucleotide Therapeutics & Delivery Follow siRNA, ASOs, splice modulation, conjugation, delivery, analytics, and manufacturing. Research questions & evidence boundaries Research guide ↗ In Vivo Cell Engineering & Gene Editing Explore in vivo CAR-T, nonviral editing deliver…
Silence Therapeutics develops siRNA medicines through its mRNAi GOLD platform. The name refers to RNA interference and gene silencing; it should not be classified as an mRNA-expression platform. Silence Therapeutics develops siRNA medicines through its mRNAi GOLD platform. The name refers to RNA int…
Oligonucleotide Therapeutics & Delivery
…ctober 27, 2026 update describes WVE-007 as an investigational GalNAc-siRNA and WVE-006 as an investigational RNA-editing oligonucleotide. These are distinct modalities, not mRNA products. oligonucleotides-wave-life-sciences-0 Corporate information Company entity Wave Life Sciences Headquarters / pr…
Oligonucleotide Therapeutics & Delivery
… registrational WVE-006 RNA-editing study in AATD and ongoing WVE-007 siRNA Phase 2a work. The company also reports FDA support for its LC-MS assay in the planned WVE-006 program. Regulatory feedback and trial plans do not constitute marketing approval or comparative efficacy evidence. Wave季度更新介绍AAT…
Oligonucleotide Therapeutics & Delivery · Bioanalysis & Biomarkers
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.
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.
An AAPS Open perspective surveys conjugation strategies for antisense oligonucleotides and siRNAs, including small-molecule ligands, cell-penetrating peptides, antibodies, and aptamers. The abstract contrasts clinically established GalNAc delivery with emerging approaches to extrahepatic tissues and discusses translation challenges. This is a synthesis of delivery platforms and clinical progress, rather than a new experimental or clinical efficacy study.
Wave's quarterly update describes planning for a registrational WVE-006 RNA-editing study in AATD and ongoing WVE-007 siRNA Phase 2a work. The company also reports FDA support for its LC-MS assay in the planned WVE-006 program. Regulatory feedback and trial plans do not constitute marketing approval or comparative efficacy evidence.