Developing a Synaptic Regenerative Therapy in ALS Using a Novel Spinogenic Small Molecule

Time: 4:00 pm
day: Day Two


  • ALS is a synaptopathy that involves an early and progressive loss of glutamatergic synapses in motor and cognitive centers
  • Developing novel small molecule therapeutics for the synaptopathies that regenerate lost synapses to physiologically relevant levels and has a lead clinical candidate (SPG302) selected for ALS
  • In preclinical studies, SPG302 improved motor function and survival in an aggressive TDP43 mouse model of ALS, and led to a recovery of respiratory function in a very defined and quantitative model of cervical spinal cord injury