Wave Life Sciences to Host Analyst and Investor Virtual Event, “Towards the Clinic: Spotlight on RNA Editing for AATD,” on September 28, 2022
“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
Dr.
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 Following the live event, an archived version of the webcast will be available on the
About WVE-006
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.
About AIMers
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.
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
Investor Contact:
617-949-4827
krausch@wavelifesci.com
Media Contact:
617-949-4817
asuter@wavelifesci.com

Source: Wave Life Sciences USA, Inc.