The Genetics Podcast
EP 155: Adeno-associated virus as a delivery vector for genetic eye disease treatment, with Paul Wille of Abeona Therapeutics
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Episode notes
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