The goal of Dr. Anguela’s research has been to develop gene-based treatments for genetic diseases, with a special emphasis on the translational advancement of AAV investigational therapies for liver-targeted indications. In his early work in academia, he contributed to lay the foundation for the development of in vivo genome editing strategies targeting the liver. During his tenure at Spark Therapeutics, he spearheaded the conception and execution of the pre-clinical hemophilia programs currently in the clinic, from early discovery to proof-of- concept and IND enabling studies. He is currently based in Donostia and serves as the Deputy CSO at AskBio Europe.