Eli Lilly will develop therapies against two drug targets for amyotrophic lateral sclerosis (ALS) that were identified and validated by Verge Genomics as part of a three-year collaboration between the companies. The targets, which weren’t disclosed, were discovered with Converge, Verge’s platform that draws on human…
RAG-21, a therapy Ractigen Therapeutics is developing for amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene, has been awarded orphan drug status by the U.S. Food and Drug Administration (FDA). Orphan drug status in the U.S. is granted to products aiming to prevent, diagnose, or treat…
The ALS Association has awarded a “groundbreaking” grant to Tiziana Life Sciences to support a small, early-stage clinical trial testing foralumab nasal spray for amyotrophic lateral sclerosis (ALS). The upcoming trial intends to investigate the safety and effectiveness of two foralumab doses in 20 ALS patients. It…
Answer ALS and Cedars-Sinai have made openly available their amyotrophic lateral sclerosis (ALS) patient-based stem cell and biodata repository to researchers worldwide. The largest of its kind, the repository contains biological and clinical data from nearly 1,000 ALS patients, as well as specialized nerve cells called induced pluripotent…
A newly awarded grant from the ALS Association will support Asha Therapeutics‘ work to advance ASHA-624, a small molecule being developed for amyotrophic lateral sclerosis (ALS), into a first-in-human clinical trial that’s expected to begin early next year. The grant was through the Lawrence and Isabel Barnett…
The European Medicines Agency (EMA) has recommended that monepantel, now named NUZ-001, be granted orphan medicinal product designation to treat amyotrophic lateral sclerosis (ALS). The positive opinion will be reviewed by the European Commission, which is expected to issue a final decision in December. If granted, the designation will provide…
A Phase 2a clinical trial testing Rho kinase (ROCK) inhibitor Bravyl (oral fasudil) in people with amyotrophic lateral sclerosis (ALS) has completed patient enrollment for its high-dose group, the company said. While the REAL trial (NTC05218668) was initially slated to only test a 180 mg daily dose of…
A new biopharmaceutical company has launched in the U.S. with more than $100 million in financing and a goal to develop a genomic medicine targeting the UNC13A protein as a novel treatment for people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Trace Neuroscience kicked off its…
GM604 is an experimental therapy designed to slow disease progression in people with ALS. It was developed by Genervon to be given via injections into the bloodstream. While positive results were seen in a Phase 2a trial, its current development status is unknown.
Researchers at Northwestern University received a $7.3 million research grant from the National Institute on Aging to expand their work on NU-9, which has shown promise in amyotrophic lateral sclerosis (ALS) preclinical studies, and explore its effectiveness in other neurodegenerative diseases. The institute is a division of the National…
