Subretinal Gene Therapy Drug AGTC-501 for XLRP Phase 1/2 Multicenter Study (HORIZON): 24-Month Safety and Efficacy Results
Subretinal AGTC-501 was generally well-tolerated. Despite all participants experiencing at least one TEAE, most of these events were mild in nature, exhibited complete resolution, and were associated with the subretinal injection procedure itself rather than the study agent. The highest dose exhibited an unfavorable risk-benefit profile due to the development of RPE changes. Although this group had the highest improvement in retinal sensitivity, our team has decided not to continue this dose in future clinical trials. Preliminary efficacy was observed at the maximum tolerated dose. Further studies are warranted to assess long-term safety and efficacy of AGTC-501 for XLRP treatment.
Authors
- Darin Curtiss
- Nadia Khalida Waheed (ORCID: https://orcid.org/0000-0002-8229-7519)
- Robert A. Sisk (ORCID: https://orcid.org/0000-0003-0328-7927)
- Antonio Yaghy (ORCID: https://orcid.org/0000-0002-5054-495X)
- David G. Birch (ORCID: https://orcid.org/0000-0002-6594-2897)
- Paul Yang
- Alessandro Iannaccone (ORCID: https://orcid.org/0000-0001-5737-8424)
- Mark E. Pennesi (ORCID: https://orcid.org/0000-0002-4943-6599)
- Andreas Lauer (ORCID: https://orcid.org/0000-0001-7582-9030)
- Rajiv Anand
- JungAh Jung
- Abraham Scaria
Institutions
- Oregon Health & Science University (US)
- Duke University (US)
- Beacon College (US)
- Cincinnati Eye Institute (US)
- Retina Foundation of the Southwest (US)
- ThermoGenesis (United States) (US)
- Astellas Pharma (United States) (US)
- Texas Retina Associates (United States) (US)
- Annie E. Casey Foundation (US)
Publication Details
- Journal
- American Journal of Ophthalmology
- Published
- 2024-12-04
- DOI
- https://doi.org/10.1016/j.ajo.2024.11.021
- Citations
- 18
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 4.21