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TDI 202

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). 


Type: Gene Therapy
Stage: Preclinical
Status: Active
Sponsor: ALS Therapy Development Institute

TDI Says:

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.

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