Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
4D-310 · 2 trials · 1 indication
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
| Arm | Type | Description |
|---|---|---|
| 4D-310 Dose Level -1 | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level -1 |
| 4D-310 Dose Level 2 | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 2 |
| 4D-310 Dose Level 3 | EXPERIMENTAL | Single IV administration of 4D-310 at Dose Level 3 |
| 4D-310 Dose Level 1 (No longer enrolling) | EXPERIMENTAL | No longer enrolling - Single IV administration of 4D-310 at Dose Level 1 |
| 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 1 - AAV NAb Titer Group A patients |
| 4D-310 Dose Level 1 - AAV NAb Titer Group B | EXPERIMENTAL | Single IV administration of 4D-310 Dose Level 1 - AAV NAb titer Group B patients |
| 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B | EXPERIMENTAL | Single IV administration of 4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients |
| 4D-310 Dose Expansion | EXPERIMENTAL | Dose expansion cohort of single IV administration of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients |
| Name | Type | Description |
|---|---|---|
| 4D-310 | BIOLOGICAL | Single IV administration of 4D-310 |
Inclusion Criteria: 1. Male or female ≥ 18 to ≤65 years of age 2. Pathogenic GLA mutation consistent with Fabry Disease 3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement 4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT...
4D-310 is an investigational gene therapy being developed for Fabry disease, a rare inherited lysosomal storage disorder. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The therapy is designed to address the underlying genetic cause of the disease.
4D-310 is being developed by 4D Molecular Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol FDMT. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational gene therapy for Fabry disease.
4D-310 is currently in Phase 1 clinical development. It is an investigational gene therapy for Fabry disease and has not been approved by the FDA or any other regulatory agency. The drug is being studied in early-stage clinical trials to assess its safety, tolerability, and potential efficacy.
4D-310 is being evaluated in two Phase 1 clinical trials. NCT04519749 is an open-label, Phase 1/2 trial in adults with Fabry disease in the United States, with 18 participants. NCT05629559 is a Phase 1 trial in adults with Fabry disease and cardiac involvement in Australia and Taiwan, also with 18 participants.
No, 4D-310 is not a monoclonal antibody. It is a gene therapy being developed for Fabry disease. The drug is designed to deliver a functional gene to address the underlying cause of the disease, rather than targeting a specific protein like an antibody would.