Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy

Yusuf, Imran H.; MacLaren, Robert E.

doi:10.1101/cshperspect.a041279

Cold Spring Harb Perspect Med

2023

DOI: 10.1101/cshperspect.a041279

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Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy

Imran H. Yusuf

Robert E. MacLaren

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Cited by 4 publications

References 81 publications

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Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

MacLaren,

Fischer,

Gow

et al. 2023

Nat Med

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Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 1011 vector genomes (vg); n = 69) or low-dose (1.0 × 1010 vg; n = 34) subretinal injection of the AAV2-vector-based gene therapy timrepigene emparvovec versus non-treated control (n = 66). Most treatment-emergent adverse events were mild or moderate. The trial did not meet its primary endpoint of best-corrected visual acuity (BCVA) improvement. In the primary endpoint analysis, three of 65 participants (5%) in the high-dose group, one of 34 (3%) participants in the low-dose group and zero of 62 (0%) participants in the control group had ≥15-letter Early Treatment Diabetic Retinopathy Study (ETDRS) improvement from baseline BCVA at 12 months (high dose, P = 0.245 versus control; low dose, P = 0.354 versus control). As the primary endpoint was not met, key secondary endpoints were not tested for significance. In a key secondary endpoint, nine of 65 (14%), six of 35 (18%) and one of 62 (2%) participants in the high-dose, low-dose and control groups, respectively, experienced ≥10-letter ETDRS improvement from baseline BCVA at 12 months. Potential opportunities to enhance future gene therapy studies for choroideremia include optimization of entry criteria (more preserved retinal area), surgical techniques and clinical endpoints. EudraCT registration: 2015-003958-41.

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Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

MacLaren,

Fischer,

Gow

et al. 2023

Nat Med

Self Cite

View full text Add to dashboard Cite

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An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study

MacLaren,

Audo,

Fischer

et al. 2024

Human Gene Therapy

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Overview of Retinal Gene Therapy: Current Status and Future Challenges

Bennett

2022

Cold Spring Harb Perspect Med

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Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy

Cited by 4 publications

References 81 publications

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study

Overview of Retinal Gene Therapy: Current Status and Future Challenges

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