Reimagining RNA medicine

CAMBRIDGE, Mass., Sept. 15, 2020 (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, announced today that it will highlight its ADAR (adenosine deaminases acting on RNA)-mediated RNA editing technology (“ADAR editing”) and its neurology programs in Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) at upcoming conferences, including the TIDES: Oligonucleotide and Peptide Therapeutics 2020 meeting and the 16^th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), taking place on September 15-18, 2020 and September 27-30, 2020, respectively.

At TIDES and OTS, Wave will share how it is developing ADAR editing as a potentially best-in-class RNA editing approach with applicability across multiple genetic diseases. Notably, one of Wave’s presentations at TIDES will include the company’s first ADAR editing data in ex vivo retina samples from non-human primates (NHPs). These data add to the growing body of in vitro and in vivo data the company has generated, which include editing in the liver of NHPs and the central nervous system in a humanized mouse model, in support of ADAR editing as an emerging platform capability at Wave.

Another presentation at TIDES will highlight Wave’s neurology pipeline, including its C9orf72 variant-selective silencing program for ALS and FTD, as well as its SNP3 program for HD, both of which are expected to initiate clinical development with the submission of clinical trial applications in the fourth quarter of 2020.

Details of Data Presentations

TIDES: Oligonucleotide and Peptide Therapeutics 2020 meeting

• “Stereopure oligonucleotides produce potent and durable activity in the eye supporting their development for inherited retinal diseases”: In this presentation, Michael Byrne, PhD, Director of In Vivo Biology and Ophthalmology at Wave Life Sciences, will provide an update on applying the company’s PRISM^TM platform to develop stereopure oligonucleotides for the potential treatment of inherited retinal diseases.
  -  Panel Discussion (“Preclinical and Clinical Development of Oligonucleotides”): Tuesday, September 15, 11:15 a.m.-12:00 p.m. ET
  -  On Demand Presentation – Track 1: Oligonucleotide Discovery, Preclinical and Clinical

• “Stereopure oligonucleotides in development for the treatment of genetically defined diseases”: In this presentation, Michael Panzara, MD, MPH, Chief Medical Officer and Head of Therapeutics Discovery and Development at Wave Life Sciences, will provide an overview of Wave’s neurology pipeline, including its clinical- and preclinical-stage allele-selective stereopure oligonucleotides for HD, as well as its preclinical C9orf72 variant-selective silencing program for ALS and FTD.
  -  On Demand Presentation – Track 1: Oligonucleotide Discovery, Preclinical and Clinical

16^th Annual Meeting of the Oligonucleotide Therapeutics Society

• “Stereopure oligonucleotides support efficient ADAR-mediated RNA editing in non-human primates”: In this presentation, Chikdu Shivalila, PhD, Senior Scientist II, Biology, at Wave Life Sciences, will provide an overview of Wave’s ADAR editing modality, which uses oligonucleotides to elicit A-to-I RNA base editing with endogenous ADAR enzymes, with particular focus on the company’s proof-of-concept in vivo study in the liver of NHPs.
  -  Short Talk Session IX: Preclinical Results (available On Demand)

About PRISM™
PRISM is Wave Life Sciences’ proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure oligonucleotides across multiple therapeutic modalities, including silencing, splicing and editing. PRISM combines the company’s unique ability to construct stereopure oligonucleotides with a deep understanding of how the interplay among oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacological properties. By exploring these interactions through iterative analysis of in vitro and in vivo outcomes and machine learning-driven predictive modeling, the company continues to define design principles that are deployed across programs to rapidly develop and manufacture clinical candidates that meet pre-defined product profiles.

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, the development of ADAR editing as a potentially best-in-class RNA editing approach and emerging platform capability at Wave; the potential for Wave’s preclinical data, including data from and its neurology programs in Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD); and the expected timing for the initiation of clinical development for Wave’s C9orf72 variant-selective silencing program for ALS and FTD, as well as its SNP3 program for HD. 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, 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 Contacts:
Kate Rausch
617-949-4827
krausch@wavelifesci.com

Graham Morrell
781-686-9600
gmorrell@wavelifesci.com

Media Contact:
Alicia Suter
617-949-4817
asuter@wavelifesci.com

Source: Wave Life Sciences USA, Inc.