Dewpoint Therapeutics has selected a novel small molecule designed to prevent the formation of abnormal TDP-43 protein clumps that drive nerve cell death in amyotrophic lateral sclerosis (ALS) and other related conditions. The candidate is specifically designed to target condensates, or membrane-less structures that concentrate specific molecules to…
Overactivity of a molecular system that normally helps clear unwanted proteins may contribute to nerve cell damage in certain genetic forms of amyotrophic lateral sclerosis (ALS), a new study suggests. The findings may open new avenues for treatment, researchers wrote in the study titled “Aberrant nuclear…
Mindwalk Holdings has identified and validated antibodies that selectively target an abnormal form of the TDP-43 protein, which is found in about 97% of amyotrophic lateral sclerosis (ALS) cases and is also linked to other neurodegenerative diseases. The candidates include antibodies that could potentially be given as a…
The first patient has been dosed in a Phase 2 clinical study testing RAG-17, a therapy being developed by Ractigen Therapeutics for people with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene (SOD1-ALS). The progression into the Phase 2 portion of the trial follows the…
Clene is scheduled to meet with the U.S. Food and Drug Administration (FDA) in the coming months to discuss new data and a potential path toward accelerated approval for CNM-Au8, an oral therapy being developed for amyotrophic lateral sclerosis (ALS). The company plans to argue that the…
The first participants have been dosed in an early Phase 1 clinical study that’s testing NRG5051, a first-in-class treatment candidate for amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, in healthy volunteers. Developed by NRG Therapeutics, the experimental therapy is designed to restore the health of mitochondria, known…
Note: This story has been updated Jan. 16, 2026, to correct that once the therapy is delivered, the gene, not the viral vector, remains inside the cell. The U.S. Food and Drug Administration (FDA) has granted fast-track designation to VectorY Therapeutics’ lead investigational therapy, VTx-002, to treat amyotrophic…
The ALS Association is urging members of Congress to take action to address issues with instability in leadership at the National Institutes of Health (NIH). At issue is the Trump administration’s decision not to renew the contract of Walter J. Koroshetz, MD, the director of the National Institute of…
Aperture Therapeutics said it is advancing its RNA-targeting therapy APRTX-003 as a potential treatment for amyotrophic lateral sclerosis (ALS). The therapy, currently in preclinical development, is designed to reduce the production of matrix metalloproteinase-9 (MMP-9) by targeting its template RNA molecule. When overactive, this enzyme may lead to…
Tanabe Pharma plans to establish a new U.S.-based company to hold the global rights to Radicava and Radicava ORS, two edaravone formulations approved to treat amyotrophic lateral sclerosis (ALS), which Shionogi plans to acquire and operate as a wholly owned subsidiary. “We are pleased to welcome Radicava…
