Amyotrophic lateral sclerosis (ALS), widely known as Lou Gehrig’s disease, is a rare neurological disease characterized by the progressive loss of motor neurons that control voluntary muscles in the brain and spinal cord. ALS is a fatal disease, and there is no known cure to stop or reverse its progression.

Though the exact pathophysiology of ALS is unclear, approximately 5–10% of the cases are familial and are mostly caused by genetic mutations. Environmental pollution, heavy metal poisoning, and viral infections are all possible factors.

In recent years, more than 20 genes associated with the pathogenesis of familial ALS have been identified.

Aiming at the three prevalent ALS gene mutations (SOD1, RNF, and TDP-43), GemPharmatech has developed ALS models to support drug development requirements.