Virtual Think-Tank on AAV vectors

The adeno-associated viral (AAV) vector-driven gene therapy field has experienced its fair share of ups and downs over the years but arguably, it has now reached its most significant tipping point. While clinical and commercial proof of concept have been achieved in the rare diseases space in recent times, including the landmark US FDA approvals of Zolgensma and Luxturna, lingering safety concerns and the challenges of product characterization (eg. around potency assay development) are significant obstacles that must be addressed if the future migration to larger patient populations is to be a success. In this Think-Tank, four sector experts discuss the key R&D, manufacturing, and regulatory trends and issues, sharing insights into the strategic and technological solutions required to move the AAV gene therapy field forward. 

Chris Lorenz is currently Senior Vice President of Technical Operations at Astellas Gene Therapies. Chris joined the company back in February 2016 when it was known as Audentes Therapeutics and was originally charged with starting up internal manufacturing operations. Since then, the TechOps organization has grown from a team of one to more than 250 spread across sites in California, North Carolina, and Japan, and has supported the development and manufacturing of dozens of AAV-based programs at both the preclinical and clinical stages. Prior to Astellas/Audentes, Chris worked for Grifols Diagnostics Solutions (formerly Novartis Diagnostics), and before that at Genentech. He holds a BS and MS in Chemical Engineering from Stanford University.

Santoshkumar Khatwani graduated from the University of Kentucky in 2010 with PhD in Chemistry. Furthermore, he obtained postdoctoral training at the University of Minnesota. Dr Khatwani then joined BioVision Inc. in 2012 and served under different capacities until 2017 where he oversaw the manufacture, testing and release of several recombinant protein, enzyme and assays for various metabolically important enzymes. Currently Dr Khatwani is serving as Director of Analytical Development at Sangamo Therapeutics with strong focus on developing analytical solutions and CMC in support of product development at early and late phase of the clinical development.

Mark White is the Associate Director of Biopharma Product Marketing at Bio-Rad. He has played a key role in the development of multiple core technology capabilities and assays alongside a multidisciplinary team of biologists and engineers at Bio-Rad and previously at Berkeley Lights Inc.. Mark obtained his PhD in Biomedical Sciences at the University of California, San Francisco.

Snehal Naik brings a confluence of regulatory affairs, early discovery, innovation, policy, and scientific expertise to her current hybrid role as Spark’s Head of Regulatory Policy and Intelligence, and Regulatory Strategy Leader for ocular programs. In this capacity she established the regulatory policy function at Spark and is supporting global development of gene therapies. Snehal co-chairs the regenerative medicine committee at BIO, staffs efforts at the Alliance for Regenerative Medicine and the Innovative Medicines Initiative, and is an active member of the American Society of Gene & Cell Therapy and New York Academy of Sciences. Snehal graduated summa cum laude with an AB-MA in Biology from Bryn Mawr College, and holds a PhD in Molecular Genetics and Genomics from Washington University in St. Louis where she also completed the Cancer Biology pathway with the Siteman Cancer Center.