Wave Life Sciences

GemVax & Kael's phase 2a trial for PSP drug GV1001 showed a 48% reduction in disease progression for the .56 mg group, with PSP-RS participants experiencing stabilization or improvement. Despite no statistical significance, the results support phase 3 trials, with GV1001 potentially becoming the first PSP treatment.

Day 2 of the 2024 Huntington Study Group Conference featured talks on palliative care, clinical trial challenges, and updates from various drug development companies, emphasizing the importance of early intervention and the use of biomarkers in HD treatment.

Wave Life Sciences announced financial results for Q3 2024, including first-ever RNA editing in humans with WVE-006 in AATD, and progress in three GalNAc-AIMer preclinical programs for cardiometabolic diseases. WVE-007 for obesity and WVE-N531 for DMD are advancing towards key milestones. FDA feedback on WVE-003 for HD supports potential accelerated approval. Cash and cash equivalents totaled $310.9 million as of September 30, 2024, with runway expected into 2027.

Despite decades of research, Huntington’s disease lacks a disease-modifying treatment. Clinical trials face challenges due to limited patient numbers and geographic barriers. Prilenia Therapeutics, Sage Therapeutics, uniQure, Wave Life Sciences, and Roche-Ionis are developing potential treatments, though some have faced setbacks. Prilenia’s pridopidine and Sage’s dalzanemdor showed mixed results, while uniQure’s AMT-130 and Wave’s WVE-003 demonstrated promising reductions in mutant huntingtin protein. Roche-Ionis’ tominersen faces ongoing development challenges.

New Huntington's disease therapies are nearing R&D finish lines, aiming to be the first disease-modifying treatments. Despite high-profile failures, companies like Wave, Prilenia, and uniQure are progressing towards regulatory approval. The field has gained momentum after decades of trial and error, with several mid-stage clinical trials underway. The ideal therapy would selectively lower mutant huntingtin and be widely distributed in the brain, though none currently exist.

Despite failed trials, hope is renewed in Huntington's disease treatment with a gene therapy showing 80% slowing of disease progression and another treatment seeking FDA accelerated approval.

Wave Life Sciences pioneers RNA editing to treat α-1 antitrypsin deficiency, using ADAR enzymes guided by gRNA to correct mRNA errors, potentially addressing Huntington’s, Duchenne muscular dystrophy, and obesity. RNA editing offers safety advantages over DNA editing, though challenges include specificity and delivery methods. Multiple companies are developing RNA editing technologies, attracting interest from major pharmaceutical firms.

Wave Life Sciences announces updates on its WVE-007 obesity treatment, new RNA editing programs for cardiometabolic diseases, and advancements in intracellular delivery chemistry, with plans to initiate a clinical trial for WVE-007 in 1Q 2025 and have five GalNAc-RNA editing or GalNAc-siRNA programs in the clinic by 2026.