Gene therapy has the potential to revolutionize medicine and improve the lives of millions of people. Adeno-associated virus (AAV) technology is an essential platform for gene therapies, as it has a low immunogenic profile and a diverse tropism for human tissues. However, a growing body of evidence suggests that the immune system can respond to AAV viral vectors used in gene therapies. This response is likely to be complex and multifactorial, just like the therapy itself.
Therefore, it is essential to understand how the host immune response impacts the efficacy, long-term durability, safety, immunogenicity and transgene expression potency in AAV gene therapy development.
In this webcast, industry experts will provide valuable insights into important aspects that must be discussed to move this exciting field forward.
You will learn:
• Important aspects of AAV efficacy and potency
• The significance, potential impact, and evaluation methods of the pre-existing humoral immune response to AAV vectors
• Current challenges and approaches for the innate immune response in relation to AAV
Unable to join the live event? Watch on demand. Register now to ensure that you receive information on how to gain access after the live event.
This webcast has been produced by Svar Life Science, who retails sole responsibility for content. About this content.
Speakers
Abraham Scaria, Chief Scientific Officer, Beacon Therapeutics
Abraham Scaria, PhD, brings 25 years of experience in gene therapy, ranging from discovery research to early-stage clinical trials. Abraham is currently the Chief Scientific Officer at Beacon Therapeutics. Most recently, he was CSO at AGTC leading the ophthalmology & neuroscience research programs. Prior to that, Abraham served as CSO at IVERIC Bio, Vice President & Head of Retinal Gene Editing at Casebia Therapeutics, and Head of Gene Therapy Research at Sanofi Genzyme. Abraham has over 50 publications and 20 patents and currently serves on several committees for the American Society for Gene and Cell Therapy.
Nageswara Rao Kollu, Senior Director, Apellis
Nageswara Rao Kollu, PhD, is currently a Senior Director at Apellis Pharmaceuticals, spearheading the Gene Therapy programs and leveraging cutting-edge technologies like base editing and other approaches to regulate complement activation to treat and potentially cure debilitating diseases. Nag previously served as a Principal Scientist at Sanofi, where he led AAV-based gene therapy initiatives to address life-threatening neuromuscular diseases, and at Precision BioSciences, where he contributed to preclinical validation of the ARCUS nuclease platform across various models. Nag earned his PhD. from the esteemed LV Prasad Eye Institute in India.
Boris Gorovits, VP of non-clinical and clinical bioanalysis, Regeneron
Boris Gorovits is VP of non-clinical and clinical bioanalysis at Regeneron Pharmaceuticals, Inc. After obtaining a PhD in Enzymology, Boris joined the Protein biophysics department at UT Medical Center, San Antonio. Boris’s industry experience includes working at Regeneron, Wyeth, Pfizer and Sana Biotechnology with a main focus on bioanalytical and PD support of diverse modalities of biotherapeutics, including MAbs, bispecifics, ADCs, AAVs and CAR-Ts. Boris is actively involved in industry conversations focusing on PK and immunogenicity assessments of various modalities, including gene therapy and CAR-T.
Moderator
Sarah Hiddleston, Nature Research Custom Media
Sarah Hiddleston is a freelance journalist who has worked with Nature Research Custom Media since 2015. Previously, Sarah worked for a decade in Madras (Chennai), India, specialising in health, pharmaceutical and environmental stories. Sarah holds an MA in Investigative Journalism from City University London, an MSc in Political Theory from the London School of Economics, and an undergraduate degree in History from the University of Cambridge, UK.
