Amyotrophic Lateral Sclerosis (ALS) Models

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by the degeneration of motor neurons in the brain and spinal cord. Each year, approximately 5,000 individuals are diagnosed with ALS, and the average life expectancy following diagnosis is typically 3 to 5 years. The primary clinical manifestations of ALS include the progressive loss of motor functions, such as movement, speech, and swallowing, leading to paralysis and, ultimately, respiratory failure. ALS poses a significant threat to human health, yet effective treatment strategies remain elusive. To date, only four drugs—Riluzole, Edaravone, Relyvrio, and Toferson—have been approved by the FDA for ALS treatment; however, their efficacy in halting disease progression remains limited.

At least 30 genes have been implicated in the pathogenesis of familial ALS, including SOD1, TARDBP, FUS, and C9ORF72. Aiming at the two prevalent ALS gene disruptions (SOD1 and TARDBP), GemPharmatech has developed ALS models to support related drug development.