WVE - Dosing underway in Wave Life Sciences' mid-stage WVE-004 study in neurodegenerative diseases

Wave Life Sciences (WVE) announces that multiple participants have initiated dosing in the Phase 1b/2a FOCUS-C9 clinical trial evaluating WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD). The FOCUS-C9 trial will assess the safety and tolerability of single- and multiple-ascending intrathecal doses of WVE-004 for people with C9-ALS and/or C9-FTD.Additional objectives include measurement of polyGP DPR proteins in the cerebrospinal fluid ((CSF)), plasma and CSF pharmacokinetics, and exploratory biomarkers and clinical outcomes.The trial is expected to enroll approx. 50 participants.The company anticipates generating clinical data in 2022 that will enable decision-making on next steps for the program.Amyotrophic lateral sclerosis ((ALA)) and Frontotemporal dementia ((FTD)) are fatal neurodegenerative disease. In ALS, progressive degeneration of motor neurons in the brain and spinal cord leads to the inability to initiate or control muscle movement.In FTD, progressive nerve cell loss in the brain's frontal lobes and temporal lobes

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Dosing underway in Wave Life Sciences' mid-stage WVE-004 study in neurodegenerative diseases