Director, Clinical Pharmacology BioMarin Pharmaceutical Inc. Berkeley, California
Gene therapies are a promising and rapidly evolving approach for treatment of rare diseases. This session will focus on the emerging field of gene therapy development with a focus on preclinical to clinical translation of AAV-mediated liver-directed gene therapy. An understanding of preclinical evaluations that support clinical development of gene therapies will be discussed. The session will also highlight opportunities and challenges in translating gene therapy products.
Understand preclinical to clinical translation considerations for gene therapies
Discover key preclinical evaluations that enable clinical translation and development of gene therapies
Learn about the role of a Clinical Pharmacologist or PK/PD Scientist in the development of gene therapies