EP 155: Adeno-associated virus as a delivery vector for genetic eye disease treatment, with Paul Wille of Abeona Therapeutics
October 10, 2024
This week Patrick is joined by Paul Wille, Director of Product Development at Abeona Therapeutics. They discuss the use of adeno-associated virus (AAV) as a vector for gene therapy delivery, outline its limitations and benefits, and hone in its potential in the context of genetic eye disorders. Paul also shares his journey from academia to industry and emphasizes the importance of building on existing technologies to advance the field.
Additionally, please consider joining us at our first in-person podcast event! We will be hosting a reception during the ASHG Annual Meeting in Denver, CO. The event will be held on the evening of Thursday 7 November. Please submit your information in this form (https://lu.ma/geneticspodASHG) to signify your interest in attending!
1:30 Intro to The Genetics Podcast
2:20 Welcome to Paul
2:53 Adeno-associated virus (AAV) biology and its advantages over other viral vectors
5:20 The gene therapy landscape and options for therapy development and delivery
6:49 The limitations of working with AAV to deliver gene therapy, including genome capacity and challenges with dividing cells
9:37 Why enormous bioreactors and huge volumes are necessary in the AAV manufacturing process
11:22 Why the eye is an ideal target for AAV gene therapy, and the potential of single-dose gene therapies
13:36 Advantages of AAVs in delivering therapies into multiple different cell types
15:17 Moving pre-clinical AAV delivered therapies into a clinical setting
17:52 How Abeona’s therapies aim to address different types of inheritance patterns
18:53 The current landscape of monogenic eye disease and Abeona’s approach to inherited eye conditions
21:38 Exploring current relationships between government, industry, and academia when supporting research for rare diseases
22:27 Paul’s transition from academia to industry
26:12 How the barrier between academia and industry is becoming more porous and bidirectional – and what that means for research
28:06 Paul’s enthusiasm for a cutting-edge area of precision medicine that has yet to gain mainstream attention
30:38 Closing remarks