WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…
WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS)Â associated with mutations in the C9ORF72 gene, has demonstrated proof-of-concept efficacy in cell and animal models, a new study shows. The nucleic acid-based therapy was able to “potently” reduce the toxic RNA molecules and small proteins…
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,…
An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…
Wave Life Sciences has gained additional financial support for an ongoing “basket” Phase 1b/2a clinical trial evaluating its experimental therapy, WVE-004, in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene. So-called basket trials, commonly used in the field of cancer, test…
Dosing has begun in a clinical trial of WVE-004, Wave Life Sciences‘ investigational therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with C9orf72 gene mutations. “ALS and FTD are devastating illnesses where therapeutic progress has been extremely limited. Advancing discovery and development of new treatments…
Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. WVE-004, an investigational treatment for people with amyotrophic lateral sclerosis…
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