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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 CEO Paul Bolno will participate in a fireside chat at the Chardan Virtual Genetic Medicines Conference on October 4, 2021, at 2:00 p.m. ET. A live webcast of the event will be available on the company’s Investor Relations page, and a replay will be archived for a limited time after the presentation. Wave Life Sciences focuses on developing treatments for devastating diseases using its PRISM platform, aiming for patient-focused solutions.
Wave Life Sciences has initiated dosing in its Phase 1b/2a SELECT-HD trial for WVE-003, aimed at treating Huntington's disease (HD). This investigational therapy specifically targets the mutant huntingtin protein while preserving the wild-type protein. The SELECT-HD trial is designed as global, multicenter, and placebo-controlled, expecting to enroll 36 patients. Preclinical data indicate WVE-003 demonstrates selective potency and durability, which are critical for clinical success in HD. Results are anticipated in 2022, with findings to be presented at the EHDN 2021 Remote Meeting.
Wave Life Sciences (Nasdaq: WVE) announced an upcoming analyst and investor research webcast on September 28, 2021, at 10:00 a.m. EDT. The event will showcase the company’s advances in ADAR-mediated RNA editing, particularly its alpha-1 antitrypsin deficiency (AATD) program, along with updates on its preclinical neurology portfolio and PRISM™ platform. Presentations will review data from the TIDES USA and Oligonucleotide Therapeutics Society meetings. Registration and live webcasts will be accessible on Wave's investor relations website.
Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company, announced that CEO Paul Bolno will present at the H.C. Wainwright 23rd Annual Global Investment Conference. The pre-recorded presentation will be available for on-demand viewing starting September 13, 2021, at 7:00 a.m. ET. Interested viewers can access the webcast on the company's Investor Relations page and it will be archived for 90 days post-event. Wave Life Sciences focuses on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform.
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.