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4D-110 · 1 trial · 1 indication
Frequency and severity of ocular and systemic AEs including clinically significant changes in ocular evaluations, systemic examinations and laboratory testing.
| Arm | Type | Description |
|---|---|---|
| 4D-110 Dose 1 | EXPERIMENTAL | 4D-110 IVT injection |
| 4D-110 Dose 2 | EXPERIMENTAL | 4D-110 IVT injection |
| 4D-110 Dose 3 | EXPERIMENTAL | 4D-110 IVT injection |
| Name | Type | Description |
|---|---|---|
| 4D-110 | BIOLOGICAL | 4D-110 drug product developed for gene therapy which comprises an AAV capsid variant (4D-R100) carrying a transgene encoding a codon-optimized human CHM gene. |
Key Inclusion Criteria: * Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing * Both eyes must have ≥ 34 ETDRS letters (\~20/200) Key Exclusion Criteria: * Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye *...
4D-110 is an investigational gene therapy being developed for choroideremia, a rare inherited retinal disease. It is designed to deliver a functional copy of the CHM gene to retinal cells. 4D-110 is in Phase 1 clinical development and has not been approved by the FDA.
4D-110 is being studied for the treatment of choroideremia, a rare X-linked retinal degeneration that causes progressive vision loss. It is an investigational therapy and is not yet approved for any indication. Clinical testing is limited to patients with a confirmed diagnosis of choroideremia.
4D-110 targets the CHM gene, which encodes the REP-1 protein. Loss-of-function mutations in CHM cause choroideremia. The therapy is intended to introduce a functional copy of CHM into retinal cells to address the underlying genetic defect. The CHM gene is the molecular target of the investigational product.
4D-110 is being developed by 4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company that trades on the Nasdaq under the ticker FDMT. The company is the sponsor of the Phase 1 study evaluating 4D-110 in patients with choroideremia.
4D-110 is in Phase 1 clinical development. It is an investigational therapy and has not received FDA approval. The only registered trial is a Phase 1 dose escalation study that is currently active but not recruiting participants.
4D-110 is being evaluated in one clinical trial, NCT04483440, titled Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia. The Phase 1 study is active but not recruiting, has enrolled 13 participants, is controlled, and is being conducted in the United States in male patients aged 18 years and older.