Hichem Tasfaout, PharmD, PhD

Scientific Advisor

Hichem is currently an assistant professor in Neurology at the University of Washington. Prior to his academic appointment, he was a postdoctoral fellow in Jeffrey Chamberlain’s research lab, where his work was honored as he was selected from thousands of applicants to present at the 2023 ASGCT Presidential Symposium. His research interest lies in developing novel gene therapies for muscular disorders. His research approach ranges from gene silencing (using antisense oligonucleotides or small hairpin RNA, also known as shRNA) to viral and non-viral gene replacement and gene editing using the CRISPR-Cas system. During his PhD thesis, he developed two therapeutic strategies to reduce the expression of Dynamin2 in myotubular myopathy. In a proof-of-concept study, he was able to show that both approaches prevent and/or reverse the myopathy progression. The results were patented in 2016 and led to the creation of Dynacure, a biotech company aiming to move forward to clinical trials. His current efforts focus on bioengineering tools to express large proteins that have been challenging to deliver with conventional methods. These techniques include chemically modified mRNA with liposomes and protein trans-splicing mediated by inteins. Hichem’s research is the backbone of the SIMPLI-GT™ platform technology, and DMD and dysferlinopathy programs at Kinea Bio.