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EU Awards €2.5M for Potential Vaccine for ALS Tied to C9orf72 Gene

A new €2.5 million (about $2.5 million) grant from the European Union (EU) will help advance a prototype vaccine for amyotrophic lateral sclerosis (ALS) associated with mutations in the C9orf72 gene — its most common genetic cause. The European Innovation Council EIC Transition Grant will be used to further develop the vaccine,…

WVE-004 Reduces Toxic Proteins, Early FOCUS-C9 Data Suggests

The experimental therapy WVE-004 appears to be engaging its intended target and reducing the amount of toxic proteins in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene, according to early data from the FOCUS-C9 clinical trial. The trial is currently recruiting adults,…

Biogen, Ionis Stopping BIIB078 Development Due to Lack of Benefit

Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…