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Beyond AAV 1.0

The gene therapy boom was built on AAV delivery tech. And recent clinical work has highlighted a variety of major hurdles; payload limitations, safety threats on high doses, fears associated with waning durability for drugs that intended to be priced once-and-done.

What are the leaders in the field doing to break down these barriers — and what could the future hold for AAV and beyond?

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Dr. Nicole Paulk

Dr. Nicole Paulk

Assistant Professor of AAV Gene Therapy, UCSF Department of Biochemistry & Biophysics in San Francisco

Dr. Nicole Paulk is an Assistant Professor of AAV Gene Therapy in the UCSF Department of Biochemistry & Biophysics in San Francisco. Dr. Paulk has a BS in Medical Microbiology, a PhD in AAV Gene Therapy and Regenerative Medicine from OHSU with Dr. Markus Grompe, and completed her Postdoctoral Fellowship in Human Gene Therapy with Dr. Mark Kay at Stanford University. She is a pioneer in the development of next-generation AAV gene delivery platforms and has engineered AAV vectors for gene repair and gene transfer for numerous rare diseases, utilized directed evolution to evolve capsid serotypes with novel tropisms, and has applied comparative proteomic and epigenomic approaches to interrogate challenges in vector manufacturing. Dr. Paulk’s translational research lab at UCSF develops solutions for the biggest problems in gene therapy: cost, delivery and efficacy, with a focus on treatments for rare diseases and cancer.

Geoff MacKay

Geoff MacKay

President & CEO, AVROBIO

Geoff is the president and CEO of AVROBIO, a leading clinical-stage lentiviral gene therapy company. A pioneer in cell and gene therapy, he brings a track record of successful leadership at innovative biotechs. He is the former CEO of Organogenesis Inc., the world’s leading cell therapy company. During his tenure at the helm, the company treated 1 million patients with living cell therapies, received the first approval of an allogeneic cell therapy from the FDA’s Center for Biologics Evaluation and Research and led the field of regenerative medicine. Geoff is also the founding CEO of eGenesis, a biotech dedicated to applying CRISPR Cas-9 gene editing to xenotransplantation. Earlier in his career, Geoff spent 11 years at Novartis in senior leadership positions within the global transplantation and immunology franchise. Geoff sits on the boards of Talaris Therapeutics and Satellos Bioscience. Past activities include chairman of the board of MassBio, chairman of the board of the Alliance of Regenerative Medicine, and a member of the advisory council to the Health Policy Commission for Massachusetts.

Eric Kelsic, PhD

Eric Kelsic, PhD

Co-founder and CEO, Dyno Therapeutics

Dyno Therapeutics is a Cambridge MA-based biotech company that is applying artificial intelligence (AI) and machine learning to engineer Adeno-associated Virus (AAV) capsids for effective delivery of gene therapies.

Prior to founding Dyno, Eric lead a team to develop the technology underlying Dyno’s AI-powered protein engineering platform in George Church’s lab at the Wyss Institute of Harvard Medical School. There, he measured the first comprehensive fitness landscape of the adeno-associated virus (AAV) capsid protein and co-discovered the AAV MAAP gene. He holds a PhD in Systems Biology from Harvard University and a BS in Physics from Caltech. During his PhD, he studied high-order interactions among soil microbes and developed MAGE-Seq, a method combing multiplexed genome-engineering with massively parallel phenotyping. As an undergrad he led one of the first studies of community structure on Facebook networks. He enjoys soccer, hiking, minimal techno and solving puzzles.

Geoff McDonough, M.D.

Geoff McDonough, M.D.

President and CEO, Generation Bio

Geoff McDonough, M.D., is the president and chief exeutive officer of Generation Bio. He formerly served as president and chief executive officer of Swedish Orphan Biovitrum AB (Sobi) from 2011– 2017. Prior to Sobi, he held a variety of senior roles at Genzyme Corporation, including president of Genzyme Europe and senior vice president and general manager of the global lysosomal storage disease business. Geoff has a Bachelor of Science in biology and a Bachelor of Arts in philosophy from University of North Carolina at Chapel Hill, graduating summa cum laude in both. He obtained his doctor of medicine at Harvard Medical School and completed his residency training in internal medicine and pediatrics at Massachusetts General Hospital and Boston Children's Hospital.

Kyle Blankenship
moderator

Kyle Blankenship

Senior Editor, Endpoints News

A native of Austin, Kyle previously worked as an associate editor at another leading biopharma publication and has held editor roles at two daily newspapers in Texas. In addition to a bachelor’s degree from Texas A&M University, Kyle holds a master’s degree in journalism from the University of North Texas in Denton. At Endpoints, Kyle leads the company’s manufacturing coverage as well as running point on virtual events, editing and content strategy. An avid cook and history nerd, you can find Kyle hovering over the stove—spatula in one hand and a World War II tome in the other.