CAMBRIDGE, Mass., Sept. 16, 2022 (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 it will hold an analyst and investor webcast on Wednesday, September 28, 2022. The event will highlight WVE-006, Wave’s A-to-I RNA base editing oligonucleotide (“AIMer”) candidate for the treatment of alpha-1 antitrypsin deficiency (AATD), provide perspectives from an expert in AATD, and outline future therapeutic applications of Wave’s best-in-class RNA editing capability.
“Towards the Clinic: Spotlight on RNA Editing for AATD”
- Date: Wednesday, September 28, 2022
- Time: 10:00 a.m. – 11:30 a.m. EDT
The event will feature presentations from the Wave Life Sciences management team including Paul Bolno, MD, MBA, President and Chief Executive Officer, Paloma Giangrande, PhD, VP, Platform Discovery Sciences, Biology, and Chandra Vargeese, PhD, Chief Technology Officer and Head of Platform Discovery Sciences.
Dr. D. Kyle Hogarth, Professor of Medicine in the Section of Pulmonary and Critical Care Medicine at the University of Chicago, will also present. Dr. Hogarth is a well-known clinical expert in the treatment of people living with AATD. He serves as the Director of the Alpha-1 Clinical Resource Center at the University of Chicago and was co-author of the 2016 clinical practice guidelines for AATD.
A Q&A session will follow the presentations.
A link to register for the upcoming event is available here. A live webcast of the event will be available on the investor relations page of the Wave Life Sciences corporate website at http://ir.wavelifesciences.com. Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website.
WVE-006 is a PN-modified GalNAc-conjugated investigational development candidate for the treatment of alpha-1 antitrypsin deficiency (AATD), designed to correct the mutant SERPINA1 Z allele transcript to address both liver and lung manifestations of disease. WVE-006 is a first-in-class RNA editing candidate (AIMer) and the most advanced program currently in development using an oligonucleotide to harness an endogenous enzyme for editing.
Adenosine deaminases acting on RNA (ADAR) enzymes are naturally occurring enzymes in humans which catalyze adenine (A) to inosine (I) changes in repetitive elements, microRNAs (miRNAs), and protein encoding transcripts. Wave’s A-to-I RNA base editing oligonucleotides (“AIMers”) are designed to recruit these endogenous ADAR enzymes to direct efficient and highly specific editing of RNA transcripts. Because I is read as G (guanine) by the cellular translational machinery, sequence-directed editing with ADAR has the potential to revert transcripts with single G-to-A point mutations that cause genetic diseases.
About Alpha-1 antitrypsin deficiency (AATD)
Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that is commonly caused by a G-to-A point mutation (“Z allele”) in the SERPINA1 gene, which leads to lung disease due to lack of wild-type alpha1-antitrypsin (M-AAT) in lungs and liver disease due to aggregation of misfolded Z-AAT protein in hepatocytes.
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.