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Also known as 4D-125 IVT Injection
4D-125 IVT · 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...
4D-125 IVT is an investigational therapy being developed for the treatment of X-Linked Retinitis Pigmentosa, a rare inherited retinal disease that primarily affects males. It is administered via intravitreal injection and is currently in Phase 1 clinical development.
4D-125 IVT is a monoclonal antibody designed for intravitreal delivery. It is being studied for its potential to address the underlying causes of X-Linked Retinitis Pigmentosa, though the specific molecular target has not been disclosed in available information.
4D-125 IVT is being developed by 4D Molecular Therapeutics, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol FDMT. The company is focused on developing targeted therapies for rare diseases.
4D-125 IVT is currently in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 1 trial is active but not recruiting participants.
4D-125 IVT is being evaluated in a Phase 1 clinical trial with the identifier NCT04517149. This trial is studying the drug in patients with X-Linked Retinitis Pigmentosa in the United States, with an enrollment of 21 male participants aged 12 years and older.
Yes, 4D-125 IVT is also known as 4D-125 IVT Injection. Both names refer to the same investigational drug product being developed by 4D Molecular Therapeutics for the treatment of X-Linked Retinitis Pigmentosa.