Emerging trends and challenges in AAV manufacturing for gene therapies
Mar
28
2023
On demand

Emerging trends and challenges in AAV manufacturing for gene therapies

Tuesday 08:00 PDT / 11:00 EDT / 16:00 BST / 17:00 CEST
Sponsor
Emerging trends and challenges in AAV manufacturing for gene therapies

Adeno-associated viral (AAV) vectors are the most prominent delivery vehicles being investigated for the treatment of genetic diseases. Although the successes of AAV-based gene therapies in pre-clinical and clinical studies are encouraging, limitations in vector characterization and manufacturing still pose significant challenges.

Here, a panel of experts in vector development and characterization will discuss the emerging trends and future challenges of AAV-based gene therapies. The panel will then dive deeper into analytical methods used to characterize vector critical quality attributes (CQAs) and highlight crucial areas for improvement in AAV manufacturing.

You will learn about:

  • Key considerations in AAV vector development
  • Important CQAs to characterize during vector engineering and manufacturing
  • Insights into developing and optimizing robust analytical workflows
Matthew Fuller
Matthew Fuller
Executive Director, Ultragenyx Gene Therapy - Research

Matthew Fuller is the Executive Director at Ultragenyx Pharmaceutical within the Ultragenyx Gene Therapy Research team. He is leading the Pinnacle Producer Cell Line (PCL) and Vector Engineering (VE) teams, both of which perform program and platform research to continually optimize and invest in Ultragenyx’s gene therapy platforms and programs. Specifically, the PCL team focuses on generation, characterization, and improvement of the Pinnacle system to maximize the quality and quantity of rAAV generated. The VE team focuses on optimization of vector construct regulatory elements to modulate and maximize tissue-specific expression and efficacy. Matthew received a PhD in molecular microbiology/virology from the University of Missouri and a MSPH from Tulane University.

Alix Eastham
Alix Eastham
Senior Manager, Cell Line Development, Ultragenyx Gene Therapy - Research

Alix Eastham is Senior Manager of the Cell Line Development Team at Ultragenyx Pharmaceutical within the Gene Therapy Research team. The team focuses on developing Pinnacle Producer Cell Lines (PCLs) for the various Ultragenyx gene therapy programs, including the characterization and improvement of the Pinnacle system to maximize the quality and quantity of rAAV generated. Alix received a MSc degree in Molecular Forensic Biology from SUNY Albany.

Ioannis (John) Eugenis
Ioannis (John) Eugenis
Product Manager, Bio-Techne

Ioannis (John) Eugenis is a Product Manager within the ProteinSimple brand of Bio-Techne. He leads the applications strategy of ProteinSimple’s flagship Simple Western product line and Single-Cell Western instruments. John received his PhD in Bioengineering at Stanford University, where he specialized in tissue engineering and regenerative medicine.