Table 4 Clinical trials on AMD gene therapy
From: Gene therapy for age-related macular degeneration: a promising frontier in vision preservation
Gene Therapy Trial | Year | Trial Phase | Details | Outcomes | References |
|---|---|---|---|---|---|
PEDF via AAV Vector | 2006 | 1 | Explored PEDF administration in nAMD patients. | Lower doses led to vision deterioration, while higher doses suggested disease stabilization. | [169] |
JNJ-81,201,887 (Janssen) | 2023 (Active, Not Recruiting) | 2 | Intravitreal injection of AAV2 vector with sCD59 gene for nAMD and GA. | Well-received, consistent reduction in GA lesion growth rates. | NCT05811351 |
RGX-314 for nAMD (by REGENXBIO and AbbVie) | 2023 | Various (see ClinicalTrials.gov) | AAV8 vector delivering anti-VEGF monoclonal antibody fragment. | Promising initial results; reduced injection frequency; ongoing trials. | NCT05407636 NCT04704921 NCT04514653 NCT04832724 NCT03066258 |
ADVM-022 by Adverum Biotechnologies | 2022 (Active, Not Recruiting) | 2 | AAV7m8 capsid delivering aflibercept expression cassette in nAMD patients. | Reduced need for anti-VEGF injections; eye inflammation as an adverse effect. | NCT05536973 |
FT-003 | 2023 | 1 | Not specified | Results not yet published. | NCT05611424 |
BD311 (AAV-CFI) | 2021 | Early phase 1 (Recruiting) | Not specified | Results not yet published. | NCT05099094 |