Treventis scientists Drs. Christopher Barden and Ross Mancini and SAB member Dr. Janice Robertson
attended the 31st Virtual International Symposium on ALS/MND that was held virtually on December 9-11th, 2020.
Dr. Robertson’s Ph.D. student Marc Shenouda presented ‘Identification and validation of small molecules targeting the amyloidogenic core of TDP-43 as therapeutics for ALS/FTLD’ on identifying small molecules and evaluating them in cell-free and cell-based assays. The results indicate significant activity in both kinds of assays, suggesting significant potential for Treventis compounds as anti-misfolding agents against TDP isoforms. Further work will establish a complete Target Product Profile and a target engagement model for advancement of compounds in lead optimization, ultimately aiming to provide a drug(s) for testing in the clinic.