ALS is a complex disorder which the cause(s) are not entirely understood. However, scientists at the ALS Therapy Development Institute have discovered a number of immune system related aspects of the disease for which they are screening potential treatments, including this anti-sense oligoneuclueotide (ASO).
ASOs are relatively new therapeutic strategies and delivery mechanisms. Early stage PK/PD experiments will be crucial to determine whether or not to continue into large scale efficacy studies.
Topics in the Pipeline