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Also known as 4D-125 IVT Injection, 4D-125 IVT
4D-125 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 4D-125 Dose Exploration | EXPERIMENTAL | Dose 1 and Dose 2 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1. The contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| 4D-125 Dose Expansion | EXPERIMENTAL | 4D-125 will be administered at the assigned dose level as a single dose, IVT injection on Day 1 in both adults and pediatric participants. For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. |
| Observational | OTHER | Natural History |
| Name | Type | Description |
|---|---|---|
| 4D-125 IVT Injection | BIOLOGICAL | 4D-125 drug product developed for gene therapy, which comprises an AAV capsid variant (4D-R100) carrying a codon-optimized human Retinitis Pigmentosa GTPase Regulator transgene. |
| Observational | OTHER | Natural History |
Natural History Key Inclusion Criteria: * Male, ≥ 6 years of age at the time of informed consent * Hemizygous non-syndromic RPGR mutation confirmed by genetic testing Interventional Key Inclusion Criteria: * Male, ≥12 years of age * Hemizygous non-syndromic RPGR mutation confirmed by genetic test...