Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
4D-710 · 1 trial · 1 indication
Safety and tolerability of 4D-710 following a single dose via inhalation, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
| Arm | Type | Description |
|---|---|---|
| 4D-710 Phase 1: Dose Exploration | EXPERIMENTAL | Participants who are ineligible for or intolerant of modulator therapy will receive one of various dose levels of 4D-710 to identify recommended phase 2 dose(s) for further evaluation. |
| 4D-710 Phase 2: Dose Expansion | EXPERIMENTAL | Participants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion. |
| 4D-710 Dose Exploration (Sub-Study) | EXPERIMENTAL | Participants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels. |
| Name | Type | Description |
|---|---|---|
| 4D-710 | BIOLOGICAL | 4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR). |
Key Inclusion Criteria (Primary Study): 1. 18 years and older 2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including: 1. Sweat chloride ≥ 60 mmol/L 2. Mutation Status * Bi-allelic mutations in the CFTR gene, or * Single mutation in the CFTR gene and clinica...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
4D-710 is an investigational gene therapy being developed for cystic fibrosis lung disease. It is designed to deliver a functional CFTR gene to lung cells to address the underlying genetic cause of the condition. The drug is currently in Phase 2 clinical development and has not been approved by the FDA.
4D-710 targets the CFTR gene, which is defective in cystic fibrosis. As a gene therapy, it aims to deliver a working copy of the CFTR gene to airway cells to restore function of the CFTR protein. This mechanism is intended to address the root cause of cystic fibrosis lung disease.
4D-710 is being developed by 4D Molecular Therapeutics, Inc., a biopharmaceutical company traded on Nasdaq under the ticker symbol FDMT. The company is conducting a Phase 2 clinical trial of 4D-710 in adult patients with cystic fibrosis lung disease.
4D-710 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The drug has received FDA orphan drug designation for rare pediatric disease, reflecting its potential role in treating a serious condition affecting children.
4D-710 is being studied in a Phase 2 clinical trial with the identifier NCT05248230, titled '4D-710 in Adult Patients With Cystic Fibrosis.' The trial is recruiting 30 participants aged 18 years and older in the United States. It is a controlled study, though it is not randomized or double-blinded.
4D-710 is a distinct investigational gene therapy developed by 4D Molecular Therapeutics. It is not known to be marketed under any other name. The drug is specifically designed for cystic fibrosis lung disease and is currently in Phase 2 trials.