Antisense Oligonucleotide Volanesoren lowers triglyceride levels in Familial Chylomicronemia Syndrome on September 18, 2019 BIOCHEMISTRY Clinical Trials Drug Discovery Lecture Notes +
Correlation of anti-AAV9 Preexisting antibody with In Vivo Transduction and NAGLU activity on September 17, 2019 Gene Therapy Immunogenicity Assessment +
Nonclinical Non-human Primate Studies for Hemophilia investigate impact of preexisting antibodies against AAV5 on therapeutic efficacy on September 17, 2019 Gene Therapy hemophilia Immunogenicity Assessment Viral Vectors +
Industry trends on use of surrogate biomaker as endpoint for clinical benefit on September 17, 2019 Drug Discovery +