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Discovery to hope: Advancing vector-based gene therapies for Parkinson’s disease

Webinar On Demand

Viral vector-based gene therapies represent a frontier in medical science, offering hope to millions of patients living with complex genetic disorders. Derived from viruses like adeno-associated virus (AAV), viral vectors are naturally adept at infecting cells and delivering genetic material, thus correcting the root cause of disease. This unique capability makes viral vectors invaluable tools, allowing for the precise and targeted delivery of therapeutic genes.

This webinar features industry-leading insights from Dr. Jennifer A. Johnston, Ph.D., Cofounder and CEO of NysnoBio. In the webinar, Dr. Johnston describes her team’s groundbreaking journey in developing an adeno-associated virus (AAV)-based gene therapy for Parkin-PD, a genetic form of Parkinson's disease that can affect individuals as young as 21 years old.

Key takeaways include:

  • The science and technology behind the development and testing of viral vectors
  • The advantages and efficacy of the baculovirus expression vector system in gene therapies
  • Strategies for transitioning from small-scale proof of concept to large-scale GMP manufacturing
  • How partnering with a large and experienced CDMO can help advance treatment development

Additionally, the webinar features Thermo Fisher Scientific’s Pouria Motevalian, Director of Viral Vector Process Development, speaking about our state-of-the-art manufacturing facility for viral vectors in Plainville, Massachusetts. Spanning 290,000 square feet, it excels in the manufacturing of viral vectors for both clinical and commercial applications, eliminating the need for costly and time-consuming facility-to-facility technology transfers.