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 | 9 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |