New UAB-developed gene therapy for ALS receives orphan drug designation

New UAB-developed gene therapy for ALS receives orphan drug designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS) developed at the Universitat Autònoma de Barcelona and licensed to the U.S. company Klotho Neurosciences, Inc.

Longitude Prize on ALS launched to award £7.5m to AI solutions

The Longitude Prize on ALS has launched to find AI-based approaches to help transform treatment of amyotrophic lateral sclerosis (ALS).

ALS gene therapy candidate KLTO‑202 wins FDA's orphan drug designation

KLTO‑202 is an experimental gene therapy that would deliver the instructions for a protein that may reduce inflammation and oxidative stress.