Published preclinical data demonstrate unprecedented Ago2 loading following administration of single subcutaneous siRNA dose, leading to improved potency and durability in vivo versus comparator siRNA formats
RNAi is one of multiple Wave modalities being advanced in strategic research collaboration with GSK
CAMBRIDGE, Mass., April 20, 2023 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the publication of preclinical data for the company’s small interfering RNA (siRNA) designs in the journal Nucleic Acids Research. Data reported include an in vivo study where a Wave subcutaneously administered, GalNAc-conjugated siRNA maintained approximately 80% silencing of HSD17B13 transcripts for three months in the liver of transgenic mice, while a comparator siRNA format lost silencing activity at the same timepoint. The paper, titled “Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference,” is available here.
“Wave’s experience with RNA-directed pharmacology, GalNAc conjugation and advancements in our proprietary chemistry have enabled the addition of RNA interference (RNAi) to our tools for discovery and drug development. This publication demonstrates that by applying our best-in-class oligonucleotide chemistry, including judiciously placed PN backbone chemistry modifications, we can dramatically improve potency and durability for GalNAc-conjugated siRNAs versus comparator siRNA formats. These silencing benefits appear to be driven, at least in part, by an approximate 10-fold increase in Ago2 loading resulting from our novel PN linkages,” said Chandra Vargeese, PhD, Chief Technology Officer and Head of Platform Discovery Sciences at Wave Life Sciences. “Moreover, we observed impressive silencing across multiple hepatic targets – mouse Ttr and human HSD17B13 – reinforcing the therapeutic potential of our approach to RNAi.”
“Wave has built the most versatile RNA medicines platform in the industry, with multiple modalities to optimally address disease biology. We continue to lead the field in RNA editing with our alpha-1 antitrypsin deficiency program and are also advancing a differentiated exon skipping portfolio in DMD, led by WVE-N531 for those amenable to exon 53 skipping. With RNAi, we have opened up a whole new capability set. The data in this publication, coupled with our recent unconjugated siRNA data showing 70-90% mRNA silencing and broad tissue exposure across six brain regions in mice,1 demonstrate our potential for achieving best-in-class silencing,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. “We expect our work in RNAi to deliver near-term value through collaborations, including our active research collaboration with GSK.”
Wave’s siRNA designs leverage the company’s novel, clinically validated and RNA-focused discovery and development platform. In this publication, Wave’s siRNAs are compared to reference molecules based on siRNAs from a commercial company with a clinically proven track record in RNAi. Highlights from the publication include:
1. Wave Life Sciences Fourth Quarter and Full Year 2022 Business Update available here
About Wave Life Sciences
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our belief that our experience with RNA-directed pharmacology, GalNAc conjugation and advancements in our proprietary chemistry may translate to best-in-class medicines; our belief that our siRNA designs demonstrate greater potential silencing benefits than comparator formats; our belief that judiciously placed PN backbone chemistry modifications result in improved potency and durability in vivo relative to comparator siRNA formats; and our understanding of what our published preclinical data portend for our work in other hepatic targets and its best-in-class therapeutic potential in humans. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release and actual results may differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.
Investor Contact:
Kate Rausch
617-949-4827
krausch@wavelifesci.com
Media Contact:
Alicia Suter
617-949-4817
asuter@wavelifesci.com
Vancouver, British Columbia--(Newsfile Corp. - November 22, 2024) - DEFENCE THERAPEUTICS INC. (CSE: DTC) (OTCQB:…
3,250 InsuJet Starter Packs Being Delivered to Pharmacies Across CanadaToronto, Ontario--(Newsfile Corp. - November 22,…
The new app allows users to browse services, book IV therapy, manage appointments, purchase packages,…
PHILADELPHIA, Nov. 22, 2024 /PRNewswire/ -- Independence Blue Cross (IBX) is implementing the Epic Payer Platform…
NEW YORK, Nov. 22, 2024 /PRNewswire/ -- Report with market evolution powered by AI -…
To help improve clinician workflow and visualization during endoscopic bronchoscopy procedures, the company has added…