Antisense Oligonucleotide Volanesoren lowers triglyceride levels in Familial Chylomicronemia Syndrome

Correlation of anti-AAV9 Preexisting antibody with In Vivo Transduction and NAGLU activity

Nonclinical Non-human Primate Studies for Hemophilia investigate impact of preexisting antibodies against AAV5 on therapeutic efficacy

Industry trends on use of surrogate biomaker as endpoint for clinical benefit