Pathway to Cures Welcomes Gene Therapy Expert Dr. Sylvia Fong to Scientific Advisory Group

Pathway to Cures, the venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF), has appointed Sylvia Fong, PhD, as a member of its Scientific Advisory Group. The announcement was made on July 30, 2025.

Expertise in Gene Therapy

Dr. Fong specializes in gene therapy, gene editing, and non-viral platforms. She is an independent consultant and adjunct associate professor at Queen’s University in Canada. Her extensive experience includes pioneering the development of the first approved AAV-based gene therapy for hemophilia A, guiding the program through research, regulatory approval, and ongoing life-cycle management.

Previous Roles and Contributions

Prior to her current role, Dr. Fong held key positions at BioMarin Pharmaceutical Inc., including executive director and head of non-oncology hematology research. There, she oversaw programs focused on hemophilia, von Willebrand disease, hereditary hemorrhagic telangiectasia, thalassemia, and sickle cell disease. Earlier in her tenure, she led translational development for therapies targeting metabolic, cardiovascular, and lysosomal storage disorders, advancing multiple programs from preclinical to clinical stages.

Focus on Rare Genetic Diseases

Dr. Fong’s expertise encompasses the development of biologics, gene therapies, and small molecules, with a strong emphasis on addressing unmet medical needs in rare genetic diseases. She continues to drive innovation across various therapeutic areas and drug modalities.

Scientific Advisory Group Membership

Dr. Fong joins Katherine High, MD. Haiyan Jiang, PhD; Glenn Pierce, MD, PhD; Steven Pipe, MD; Leonard Valentino, MD; and Michael Recht, MD, PhD, MBA, as members of the Pathway to Cures Scientific Advisory Group.

Further information about Dr. Fong’s appointment can be found on the NBDF website.