Gene Therapy AGTC-501 Hits Phase 1/2 Marks for Rare Pediatric Blindness


A podcast interview with study author Paul Yang, MD, PhD, on the current research and future implementation of the agent.

Episode highlights

0:17 Introduction
2:37 Dr. Paul Yang
3:17 Phase 1/2 findings for AGTC-501
7:06 The characteristics of XLRP
8:40 Other phases of research
10:06 The ocular gene therapy care team
12:01 The parent conversation
13:51 Gene therapy advocacy
16:09 Final thoughts
17:50 Outro

New phase 1/2 data presented at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Virtual Meeting this week showed investigative gene therapy AGTC-501 provided notable efficacy and safety mark benefit in patients with X-linked retinitis pigmentosa (XLRP).

The six-moth findings, presented by author Paul Yang, MD, PhD, of the Casey Eye Institute at Oregon Health & Science University, lead to hope for even greater and more concrete outcomes as the agent is assessed through 5 years in the patients aged ≥6 years old with the rare vision loss disease.

In a DocTalk podcast interview conducted during ARVO 2021, Yang joined to discuss the newest phase 1/2 findings, as well as the properties of benefit being assessed in AGTC-501.

Yang also explained the reason for expansive gene therapy research in XLRP, the role of advocacy and awareness-building in gene therapy research for rare disease, and what he would define as the modern ocular gene therapy care team.

The study, “Six Month Findings from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Show Clinically Meaningful Improvement in Macular Sensitivity,” was presented at ARVO 2021.

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