This week on The Genetics Podcast, Patrick is joined by Daniel Chung, Chief Medical Officer of Beacon Therapeutics. They discuss lessons from developing the first ocular gene therapy (Luxturna), the complexities of designing and executing pivotal trials in inherited retinal disease, and how endpoint selection, delivery, and immunogenicity shape clinical and commercial success.
Show Notes:
0:00 Intro to The Genetics Podcast
00:59 Welcome to Daniel
01:57 Daniel’s firsthand experience with the development and approval of Luxturna for inherited retinal dystrophy
06:13 Factors contributing to delays in approval for gene therapies in recent years
08:13 Overview of ocular diseases and their suitability for gene therapy
11:40 Why Daniel joined Beacon Therapeutics and current priorities for the lead program
13:22 Key challenges in designing and executing a pivotal gene therapy trial for X-linked retinitis pigmentosa (XLRP)
15:49 Lessons from prior inherited retinal disease trials and selecting the right clinical endpoints
21:03 Expanding beyond the lead program into earlier patients and additional ocular indications
23:43 AAV packaging constraints and managing immunogenicity in ocular gene therapy
26:29 Lessons from academia–industry collaboration in developing gene therapies
28:23 Access to genetic testing in inherited retinal diseases and remaining gaps
32:10 Key scientific, regulatory, and commercial challenges facing gene therapy today
35:39 Closing remarks
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