ET9 / Expression Therap - LARVOL DELTA

Humanization and functional characterization of enhanced coagulation factor IX variants identified through ancestral sequence reconstruction. (PubMed, J Thromb Haemost) - "Starting from computationally inferred ancient FIX sequences, novel amino acid substitutions conferring activity enhancement were identified and translated into an AAV-FIX gene therapy cassette demonstrating high potency. This ASR discovery and sequence mapping refinement approach represents a promising platform for broader protein drug and gene therapy candidate optimization."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases