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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 the treatment of Fabry Disease, a rare genetic disorder. It is designed to address the underlying cause of the disease by delivering a functional copy of the GLA gene to produce the missing enzyme. 4D-310 is currently in Phase 1 clinical trials.
4D-310 targets the GLA gene, which encodes the enzyme alpha-galactosidase A. In Fabry Disease, mutations in this gene lead to enzyme deficiency and harmful buildup of certain fats in cells. By delivering a working GLA gene, 4D-310 aims to restore enzyme activity and potentially alleviate disease symptoms.
4D-310 is being developed by 4D Molecular Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol FDMT. The company is focused on developing targeted gene therapies for rare diseases, with 4D-310 being one of its lead candidates for Fabry Disease.
4D-310 is currently in Phase 1 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing Phase 1 trials are designed to evaluate the safety, tolerability, and preliminary efficacy of 4D-310 in adult patients with Fabry Disease.
4D-310 is being studied in two Phase 1 clinical trials. The first, NCT05629559, is recruiting adults with Fabry Disease and cardiac involvement in Australia and Taiwan. The second, NCT04519749, is an active but not recruiting open-label trial in the United States. Both trials enroll 18 participants each.
Yes, 4D-310 is a form of gene therapy. It is designed to deliver a functional copy of the GLA gene to cells, addressing the root cause of Fabry Disease. This approach differs from traditional enzyme replacement therapies, as it aims to provide a long-term or potentially one-time treatment by restoring the body's ability to produce the needed enzyme.