Patent Title: Double Stranded Oligonucleotide Compositions for RNA Interference and Methods Relating Thereto
Publication Number: US 20250382617 A1
Filing Date: August 29, 2025
Publication Date: December 18, 2025
Applicant/Assignee: Wave Life Sciences Ltd.
What This Covers
This patent application focuses on novel double‑stranded oligonucleotide molecules and associated compositions designed to mediate RNA interference (RNAi) — a mechanism by which specific gene expression is silenced in disease‑relevant tissues.
Key aspects of the invention include:
- Structural design of oligonucleotides: The technology covers optimized structural elements — such as base sequence, sugar and base modifications, internucleotidic linkages, and patterns of stereochemistry — that significantly influence the biological performance of oligonucleotides. These structural features can improve RNAi activity, Ago2 loading, thermal stability, in vivo stability, and delivery into cells and tissues.
- Enhanced therapeutic properties: By tuning these chemical and stereochemical features, the compositions aim to increase potency, target specificity and cellular uptake while reducing off‑target effects — critical for effective RNA medicines targeting liver, central nervous system (CNS) or other tissues.
- Methods of administration and therapeutic use: The application also claims methods of using these oligonucleotide compositions to treat human diseases through RNAi‑mediated gene silencing, potentially including hepatic and CNS disorders, which are key areas of Wave’s clinical and preclinical programs.
Why This Is Important
1) Central to Wave’s RNA Medicine Platform
Wave focuses on proprietary stereopure oligonucleotide technologies as the backbone of its therapeutic pipeline, including programs for obesity, alpha‑1 antitrypsin deficiency, Huntington’s disease and other conditions. Innovations in oligonucleotide structure are foundational to improving efficacy, safety and drug delivery, and protecting these innovations is crucial to differentiating Wave’s products.
2) Supports Broad Therapeutic Potential
RNA interference is a powerful mechanism for treating diseases with well‑validated genetic targets. Better double‑stranded oligonucleotides can be applied across multiple indications, increasing the potential commercial value of this IP beyond any single drug.
3) Strengthens Competitive IP Position
Wave’s platform competes with other RNA therapeutics companies (e.g., Alnylam, Ionis). Strong patents on chemical modifications and stereochemical designs help protect exclusive rights to high‑performance oligonucleotide compositions, which can support longer product lifecycles and higher barriers to competition.
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