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…
Both people living with amyotrophic lateral sclerosis (ALS) and their caregivers benefit most from flexible, personalized psychological support that combines emotional care and open dialogue, a new study from Germany suggests. Critical, the researchers say, is to create a framework that can be easily adjusted to a patient and…
QRL-101, an experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS), was found in an early clinical trial to engage its intended biological target and reduce nerve cell overactivity in people with ALS. These results come from a proof-of-mechanism Phase 1 clinical study…
Health Canada has cleared the way for people with amyotrophic lateral sclerosis (ALS) living in Canada to join an ongoing clinical trial evaluating Coya Therapeutics’ therapy candidate COYA 302. The Canadian regulatory agency issued a “no objection” letter that will allow the developer to start enrolling adults in…
