Wave Life Sciences

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) 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,…

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…

New antisense oligonucleotide (ASO) molecular therapies targeting the most common gene mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia reduced brain disease hallmarks in a mouse study, researchers found. The study, “Stereopure Antisense Oligonucleotides Preferentially Knockdown G4C2 Repeat-Containing C9ORF72 Transcripts: A Potential Therapeutic Approach for the Treatment…

The Wave Life Sciences therapy WVE-3972-01 reduced markers of the form of ALS that scientists believe stems from a gene mutation, studies show. Researchers said the potential treatment blocked faulty versions of messenger RNA associated with the production of abnormal C9orf72 protein. The RNA is an intermediary between the C9orf72 gene…

Wave Life Sciences is developing two ALS and frontotemporal dementia therapies that target a gene mutation common in both. The therapies target mutations of the C9orf72 gene, Wave said in a business update. “I am excited to announce our next development program targeting C9orf72 mutations in ALS and FTD,” Dr.