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Also known as VX-522 mRNA therapy
VX-522 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Single Ascending Dose (SAD) | EXPERIMENTAL | Participants grouped into different cohorts will receive a single ascending dose of VX-522. |
| Multiple Ascending Dose (MAD) Cohort 1: VX-522 | EXPERIMENTAL | Participants will receive multiple ascending doses of VX-522. |
| MAD Cohort 1: VX-522+ IVA | EXPERIMENTAL | Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. |
| MAD Cohort 2: VX-522+ IVA | EXPERIMENTAL | Following run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA. |
| Name | Type | Description |
|---|---|---|
| VX-522 mRNA therapy | DRUG | Oral inhalation using nebulizer. |
| IVA | DRUG | Tablet for oral administration. |
Key Inclusion Criteria: * Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2) * A total body weight greater than (\>) 50 kg * Stable CF disease * CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy o Example mutations include but are not ...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-272, Tezacaftor |
| 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
VX-522 is an investigational RNA therapy being developed for cystic fibrosis, a genetic disease affecting the lungs and other organs. It is designed as an inhaled treatment for people with CF. It has been studied in a Phase 1/2 clinical trial in adults with cystic fibrosis.
VX-522 targets CFTR, the gene that is defective in cystic fibrosis. It is an mRNA therapy designed to deliver genetic instructions for producing functional CFTR protein. By targeting the underlying genetic cause, it aims to address the disease at its source rather than only managing symptoms.
VX-522 is being developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is a biopharmaceutical company focused on serious diseases, including cystic fibrosis, and VX-522 is part of its pipeline of investigational CF therapies.
VX-522 is in Phase 1 clinical development. It has completed one Phase 1/2 trial in participants with cystic fibrosis. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency for any indication.
VX-522 has been studied in a single Phase 1/2 trial, NCT05668741, titled A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis. The trial enrolled 26 adults across the United States, Australia, Belgium, Canada, Germany, Sweden, and the United Kingdom, and it is now completed.
Yes, VX-522 mRNA therapy is another name for VX-522. Both refer to the same investigational inhaled mRNA treatment for cystic fibrosis being developed by Vertex Pharmaceuticals. The mRNA designation reflects its mechanism of delivering messenger RNA to cells to produce functional CFTR protein.