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Wave Life Sciences Ltd. (Nasdaq: WVE) is a clinical-stage biotechnology company harnessing its groundbreaking synthetic chemistry platform to create stereopure nucleic acid therapeutics. Focused on rare genetic diseases, Wave's pipeline addresses conditions such as alpha-1 antitrypsin deficiency, obesity, Duchenne muscular dystrophy (DMD), and Huntington's disease. Wave's proprietary PRISMTM platform integrates various RNA modalities, including RNA editing, antisense, exon-skipping, and RNA interference, enabling scientific breakthroughs that address both rare and common disorders.
Recently, Wave has made significant progress with its Phase 2 FORWARD-53 trial for DMD, showing promising results with WVE-N531. This trial, which began dosing in December 2023, aims to evaluate dystrophin protein generation in boys amenable to exon 53 skipping, with critical data expected in 2024. The company also anticipates delivering first-of-its-kind RNA editing proof-of-mechanism data in 2024 for its WVE-006 program, targeting alpha-1 antitrypsin deficiency.
Financially, Wave is well-capitalized, with substantial cash inflows and strategic partnerships, including a notable collaboration with GSK. This partnership has led to significant milestone payments and the initiation of development programs utilizing Wave's advanced GalNAc-siRNA format.
Wave Life Sciences is at the forefront of RNA medicine, driven by a commitment to transform human health and provide life-changing treatments for devastating diseases.
Wave Life Sciences (Nasdaq: WVE), a clinical-stage genetic medicines company, announced that Paul Bolno, MD, CEO, will participate in two investor conferences in May 2022. The first is at the RBC Capital Markets 2022 Global Healthcare Conference on May 17, 2022, from 2:05 p.m. to 2:30 p.m. ET in New York. The second is at the H.C. Wainwright Global Investment Conference on May 24, 2022, from 3:30 p.m. to 4:00 p.m. ET in Miami. Live webcasts will be available on their Investor Relations page.
Wave Life Sciences Ltd. (Nasdaq: WVE) has announced a live webcast and conference call scheduled for May 12, 2022, at 8:30 a.m. ET to discuss its first quarter 2022 financial results and provide a business update. The event will be accessible via the company's investor relations website, with teleconference options available for domestic and international participants. Wave Life Sciences focuses on developing life-changing genetic medicines using its proprietary PRISM platform to target genetically defined diseases.
Wave Life Sciences announced the publication of preclinical data for WVE-004, targeting C9orf72-associated ALS and frontotemporal dementia. The study, published in Molecular Therapy Nucleic Acids, demonstrated significant reductions in pathogenic RNA transcripts and poly(GP) proteins over six months in mice, highlighting its potential efficacy. Initial clinical data from the ongoing FOCUS-C9 trial suggests that these preclinical results are translating into human applications, with effective target engagement and sustained reductions in poly(GP) levels.
Wave Life Sciences Ltd. (Nasdaq: WVE) will participate in Chardan’s 6th Annual Genetic Medicines & Cell Therapy Manufacturing Summit on April 26, 2022, at 2:30 p.m. ET. CEO Paul Bolno and SVP Sri Vaddeboina will join an analyst-led fireside chat, which will be streamed live on their Investor Relations page. Wave Life Sciences focuses on delivering innovative genetic medicines for devastating diseases using its PRISM platform for the precise design and production of stereopure oligonucleotides. A replay will be available shortly after the event on their website.
Wave Life Sciences (Nasdaq: WVE) announced positive updates from its Phase 1b/2a FOCUS-C9 trial for WVE-004, targeting C9orf72-associated ALS and frontotemporal dementia. Key findings include significant reductions in poly(GP) levels indicating target engagement, with a 34% decrease observed post a 30 mg single dose. The dosing observation period will now extend to six months to assess maximum poly(GP) reduction. Multidose cohorts at 10 mg are underway, with further data expected throughout 2022, reinforcing the trial’s adaptive design and the potential of its proprietary PN chemistry.
Wave Life Sciences Ltd. (Nasdaq: WVE) announced that CEO Paul Bolno will participate in a virtual fireside chat at the Stifel 2022 CNS Days event on March 28, 2022, at 9:15 a.m. EDT. This event highlights Wave's commitment to developing genetic medicines aimed at treating severe diseases. A live webcast of the presentation will be available on the Investor Relations page of Wave's website, with a replay available for a limited time after the event.
Wave Life Sciences Ltd. (Nasdaq: WVE) announced key presentations at the OPT Congress and RNA Editing Summit, showcasing advancements in their A-to-I(G) RNA base editing oligonucleotides (AIMers). Highlights include preclinical data on their AIMer program for Alpha-1 antitrypsin deficiency (AATD), demonstrating editing efficiency and protein restoration over 19 weeks. The AIMers leverage the body's natural ADAR enzymes for precise editing without permanent genomic changes. The company's IND-enabling studies are slated for Q3 2022, emphasizing Wave's commitment to innovative genetic therapies.
Wave Life Sciences (Nasdaq: WVE) announced preclinical proof-of-concept data demonstrating the efficacy of its AIMer technology for RNA base editing in non-human primates. Published in Nature Biotechnology, the study reported up to 50% editing of the ACTB transcript in the liver, with levels persisting at 40% for over a month. AIMers simplify delivery without the need for exogenous enzymes, potentially enhancing therapeutic applications, especially for genetic conditions like alpha-1 antitrypsin deficiency.
Wave Life Sciences (WVE) reported a net loss of $34.8 million for Q4 2021, compared to $28.8 million in Q4 2020. For the full year, the net loss was $122.2 million, down from $149.9 million in 2020. Revenue from the Takeda collaboration fell to $1.8 million in Q4 from $9.4 million in the previous year, while total revenue for 2021 reached $41.0 million, up from $20.1 million. Cash as of December 31, 2021, stood at $150.6 million, expected to fund operations into Q2 2023. Key developments include advancements in RNA therapies for ALS, DMD, and HD.
Wave Life Sciences (Nasdaq: WVE) will hold a live webcast and conference call on March 3, 2022, at 8:30 a.m. ET to discuss its Q4 and FY 2021 financial results and provide a business update. The webcast can be accessed via the Investor Relations section of the Wave Life Sciences website. The company's proprietary PRISM platform focuses on developing genetic medicines, targeting genetically defined diseases. Investors can dial in for the call using provided numbers and enter the conference ID: 7694386. An archived version will be available post-event.