Wave Life Sciences Ltd.

We are a clinical-stage biotechnology company focused on developing ribonucleic acid ("RNA") medicines, also known as oligonucleotides, or those targeting RNA, to improve human health. Our PRISM® RNA medicines platform integrates multiple approaches, chemistry innovations, and insights from human genetics to create scientific advancements for both rare and common disorders. Our platform includes RNA-targeting methods such as RNAi (SpiNA) and RNA editing (AIMers). Our pipeline includes programs for obesity (WVE-007), alpha-1 antitrypsin deficiency ("AATD") (WVE-006), and PNPLA3 I148M liver disease (WVE-008). We also have clinical programs for Duchenne muscular dystrophy ("DMD") and Huntington's disease ("HD"), along with several preclinical programs. We were founded over a decade ago to use chemistry innovation to adjust the pharmacological properties of oligonucleotides. Our chemistry modifications have been observed in clinical trials to enhance the potency, distribution, and durability of our molecules. Our chemistry allows us to use clinically proven conjugates, such as N-acetylgalactosamine ("GalNAc"), or free uptake for delivery to various cell and tissue types, rather than complex delivery vehicles like lipid nanoparticles and viruses. We hold intellectual property for our novel chemistry modifications. Our chemistry capabilities have also enabled new biological applications, including using adenosine deaminases acting on RNA ("ADAR") enzymes for messenger RNA ("mRNA") correction and upregulation, and selectively silencing a mutant allele.