Business
Wave Life Sciences Highlights Pipeline Progress and Expansion Leveraging New PN Backbone Chemistry Modifications
Three clinical trials to begin in 2021 with compounds containing Wave’s novel PN backbone chemistry modifications Data from ongoing PRECISION-HD and OLE
About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Three clinical trials to begin in 2021 with compounds containing Wave’s novel PN backbone chemistry modifications Data from ongoing PRECISION-HD and OLE clinical trials for Huntington’s disease expected by end of 1Q 2021 Potential best-in-class ADAR editing platform capability continues to advance, with validation of proprietary in vivo modeling system and delivery of in vivo alpha-1 antitrypsin deficiency data expected 1H 2021 CAMBRIDGE, Mass., Jan. 11, 2021 (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 key upcoming milestones for 2021, including the initiation of new clinical trials, expected data readouts, and continued advancement of Wave’s proprietary discovery and drug development platform, PRISM™. “2020 was a year of focused and formative progress for Wave, which culminated with submissions of clinical trial applications for two new programs. We continued to deliver on our ambitious goals despite the pandemic and are now on a course to unlock significant value from our pipeline and platform starting in 2021. Our research and clinical teams have made impressive headway across our portfolio of investigational stereopure oligonucleotides, and today we are advancing more than a dozen silencing, splicing and editing programs across various stages of development,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “This year, we plan to initiate clinical trials for three compounds containing PN backbone chemistry modifications, which have been shown preclinically to increase potency, exposure and durability across various modalities. With our three new trials, we’ll be able to more fully assess the potential of this novel chemistry advancement for the field of genetic medicine. They also offer the opportunity to deepen our impact in Huntington’s disease and extend our research to others struggling with amyotrophic lateral sclerosis, frontotemporal dementia, and neuromuscular diseases.” “We also plan to deliver comprehensive data results from the ongoing PRECISION-HD trials late in the first quarter to enable a decision regarding potential Phase 3 development for WVE-120101 and WVE-120102, our first-generation Huntington’s disease ca...