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?
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 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.
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.