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VX-522 mRNA therapy

Phase 1

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: May 22, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment26

FDA Designations

No designations recorded

Clinical trial landscape

VX-522 mRNA therapy · 1 trial · 1 indication

Phase 1 1
NCT05668741A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)Cystic Fibrosis
ACTIVE NOT_RECRUITING26 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Cystic Fibrosis
NCT0566874126
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Study Endpoints

Primary Endpoints

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Week 8 [SAD and MAD]

Secondary Endpoints

MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline at Day 29
MAD: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 Through Safety Follow-up Visit [up to Week 28]
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Single Ascending Dose (SAD)EXPERIMENTALParticipants grouped into different cohorts will receive a single ascending dose of VX-522.
Multiple Ascending Dose (MAD) Cohort 1: VX-522EXPERIMENTALParticipants will receive multiple ascending doses of VX-522.
MAD Cohort 1: VX-522+ IVAEXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.
MAD Cohort 2: VX-522+ IVAEXPERIMENTALFollowing run-in period with ivacaftor (IVA), participants will receive multiple ascending doses of VX-522 with IVA.

Interventions

NameTypeDescription
VX-522 mRNA therapyDRUGOral inhalation using nebulizer.
IVADRUGTablet for oral administration.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites26

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 ...

Countries:United StatesAustraliaBelgiumCanadaGermanySwedenUnited Kingdom
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Competitive Landscape -Cystic Fibrosis 17 trials

CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX8PHASE3VX-121/TEZ/D-IVA
Sionna Therapeutics, Inc.SION2PHASE2SION-719
BiomX Inc.PHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 - Undisclosed
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Frequently asked questions about VX-522 mRNA therapy

What is VX-522 used for?

VX-522 is an investigational mRNA therapy being developed for the treatment of cystic fibrosis. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The therapy is designed to address the underlying cause of the disease in patients with this condition.

Who makes VX-522?

VX-522 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for cystic fibrosis.

What phase is VX-522 in?

VX-522 is currently in Phase 1 clinical development. It is an investigational therapy, meaning it has not been approved by regulatory agencies. The ongoing clinical trial is designed to assess the safety, tolerability, and potential efficacy of the treatment in patients with cystic fibrosis.

What clinical trials is VX-522 in?

VX-522 is being studied in a Phase 1/2 clinical trial identified as NCT05668741. This trial is currently active but not recruiting participants, with an enrollment of 26 individuals. The study is being conducted across multiple countries, including the United States, Australia, Belgium, Canada, Germany, Sweden, and the United Kingdom.

Is VX-522 the same as other cystic fibrosis treatments?

VX-522 is a distinct investigational mRNA therapy developed by Vertex Pharmaceuticals for cystic fibrosis. It is not the same as other approved treatments, as it is still in clinical development. The therapy is being evaluated in a controlled clinical trial to determine its safety and effectiveness in patients.