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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 Ltd. (Nasdaq: WVE) announced it will present nine findings at TIDES USA and the 17th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS). These conferences will showcase advancements in ADAR-mediated RNA editing and the PRISM™ platform. Significant presentations include enhancing pharmacologic profiles of CNS-targeted therapies and a program for alpha-1 antitrypsin deficiency. The events will occur from September 20-29, 2021, further emphasizing Wave's commitment to developing precise treatments for genetically defined diseases.
Wave Life Sciences (WVE) has initiated the FOCUS-C9 clinical trial for WVE-004, targeting C9orf72-associated ALS and FTD. The company reported a net loss of $38.8 million for Q2 2021, slightly down from $40.5 million in Q2 2020. R&D expenses remained stable at $31.6 million. A notable highlight includes proof-of-concept data showcasing over 90% knockdown of polyGP proteins. Wave anticipates generating significant clinical data through 2022 and aims to utilize its PRISM platform for advancements in various genetic diseases. Cash and equivalents totaled $143.8 million as of June 30, 2021.
Wave Life Sciences Ltd. (Nasdaq: WVE) announced a live webcast on August 5, 2021, at 8:30 a.m. ET to discuss its Q2 2021 financial results alongside a business update. The call will be accessible via the Investor Relations page on their website or by phone using specific dial-in numbers. The company focuses on developing genetic medicines and aims to deliver best-in-class treatments for genetically defined diseases using its proprietary PRISM platform. For more information, visit Wave's website.
Wave Life Sciences announced the first clinical dosing of WVE-004 in the Phase 1b/2a FOCUS-C9 trial for C9-ALS and C9-FTD, targeting a genetic cause of these diseases. The trial aims to optimize dosing adaptively, with results anticipated in 2022. Preclinical studies showed a robust knockdown of harmful proteins and promising pharmacological properties of WVE-004, enhancing Wave's drug development strategy through its novel PN chemistry. Approximately 50 participants are expected to enroll, focusing on safety and efficacy.
Wave Life Sciences (Nasdaq: WVE) announced the first proof-of-concept in vivo data for its ADAR-mediated RNA editing program targeting alpha-1 antitrypsin deficiency (AATD). The study showed up to 40% editing of the SERPINA1 Z-allele mRNA in the liver, resulting in a significant increase in functional AAT protein. The data suggest a promising approach to address both lung and liver disease manifestations by reducing mutated protein aggregation. Wave plans to present further in vivo data in late 2021 as it progresses towards clinical applications.
Wave Life Sciences (Nasdaq: WVE) announced that CEO Paul Bolno will present at the Jefferies Virtual Healthcare Conference on June 2, 2021, at 11:30 a.m. ET. The presentation will include a corporate update and discuss in vivo data from the company’s ADAR editing discovery program targeting alpha-1 antitrypsin deficiency (AATD). A live webcast will be available on their investor relations page, with a replay accessible for 90 days post-event. Wave Life Sciences is focused on developing genetic medicines for severe diseases.
Wave Life Sciences Ltd. (WVE) reported a net loss of $42.5 million for Q1 2021, an improvement from $47.5 million in the same quarter of 2020. R&D expenses fell to $33.4 million due to the wind-down of the suvodirsen program. The company ended the quarter with $148.5 million in cash, down from $184.5 million at the end of 2020. Significant clinical updates include advancements in programs for ALS, Huntington's disease, and Duchenne muscular dystrophy. Wave's collaboration with Takeda is expected to provide $30 million in support, ensuring funding into Q2 2023.
Wave Life Sciences (Nasdaq: WVE) announced that CEO Paul Bolno will present at the RBC Capital Markets Global Healthcare Virtual Conference on May 19, 2021, at 4:15 p.m. ET. This presentation will be streamed live and can be accessed via the company's investor relations page. Following the event, a replay will be available for 90 days. Wave Life Sciences is dedicated to developing innovative genetic medicines to treat severe diseases, utilizing its proprietary discovery platform, PRISM.
Wave Life Sciences Ltd. (Nasdaq: WVE) will host a live webcast and conference call on May 13, 2021, at 8:30 a.m. ET to discuss its Q1 2021 financial results and provide a business update. This event will be accessible via their Investor Relations page. As a clinical-stage genetic medicines company, Wave focuses on innovative treatments for serious diseases through its proprietary PRISM platform that enhances the design and production of stereopure oligonucleotides.
Wave Life Sciences Ltd. (Nasdaq: WVE) has announced results from its PRECISION-HD trials, revealing no significant efficacy for investigational treatments WVE-120102 and WVE-120101 in reducing mutant huntingtin protein (mHTT) in Huntington's disease patients. Consequently, clinical development for both candidates will be halted. However, the company is advancing its novel WVE-003 program with improvements in pharmacology and allele-selectivity targeted at SNP3. Wave plans to initiate dosing for WVE-003 in 2021, showcasing commitment to developing therapies that spare healthy wild-type huntingtin protein.
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