A study to evaluate a new mRNA therapy in adults with CF mutations that don’t respond to CFTR modulator therapies (VX21-522-001)


CTAP badge
Therapeutic approach
Genetic Therapy
Trial status
Open to recruitment Participating Centres
Trials Tracker ID
Last updated

Full title

A phase 1 study to evaluate the safety, tolerability and effectiveness of VX-522 mRNA therapy in people with CF aged 18 and over with CFTR mutations that don’t respond to CFTR modulator therapies.

Study details

mRNA therapy is a new type of treatment for people with CF, to be assessed in clinical trials.

If you are aged between 18 and 65 and have CF mutations that mean you can’t take CFTR modulator treatments, you may be eligible to take part in this study. Example CF mutations could include Class I mutations (where no CFTR protein is produced), nonsense mutations (like G542X and W1282X), and some types of splicing mutations. Talk to your CF team if you don’t know which CF mutations you have or if you think you might be eligible.

This study – also referred to as the BEACON-CF clinical research study – is exploring the safety and tolerability of a single dose of VX-522 (an inhaled mRNA therapy) for people with cystic fibrosis who are not expected to benefit from CFTR modulators.

Participants will be given a single dose of VX-522, which they will inhale through a nebuliser.

Participants will be asked to take part in daily monitoring visits for the first week. They will also be asked to attend follow-up visits on day 14, day 28, week 12, and week 24.

Find out more about  genetic therapies and what’s involved in genetic therapy clinical trials.

Please note that you do not need to be receiving your usual care from one of the recruiting centres to be able to take part in the trial. Please speak to your local CF team or use the ‘Get in touch’ button opposite if you are interested in taking part.

Study updates

Screening for the Multiple Ascending Dose (MAD) part of this trial has now begun. 

Phase 1
Length of participation
28 weeks
CF sponsor

Who can take part?

Age range
Between 18 and 65 years
Including people

With two CFTR gene mutations that are not responsive to CFTR modulator treatments like Kaftrio, Orkambi and Symkevi

FEV1 greater than or equal to 40% of predicted mean for age, sex, and height

Total body weight greater than 50 kg

Excluding people

With a history of uncontrolled asthma within a year prior to screening

History of solid organ or hematological transplantation

Talk to your CF team if you’re not sure whether you’re eligible.

Get in touch

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CF centres running this trial


Cambridge Royal Papworth Hospital (Adults)

CTAP centre
NHS Trust
Royal Papworth Hospital NHS Foundation Trust
Papworth Road
Trial Coordinators
Deepa George
Victoria Christenssen

Glasgow - Queen Elizabeth University Hospital

CTAP centre
NHS Trust
NHS Greater Glasgow & Clyde
1345 Govan Road
G51 4TF
Trial Coordinators
Annie Husband

Manchester (Adults) - Wythenshawe Hospital

CTAP centre
NHS Trust
Manchester University NHS Foundation Trust
Wythenshawe Hospital
South Moor Road
M23 9LT
Trial Coordinators
Cindy Chan

Royal Brompton Hospital (London)

CTAP centre
NHS Trust
Guy's and St Thomas' NHS Foundation Trust
Royal Brompton Hospital
Sydney Street
Trial Coordinators
Yasmine Needham

Southampton General Hospital

CTAP centre
NHS Trust
University Hospital Southampton NHS Foundation Trust
Tremona Road
SO16 6YD
Trial Coordinators
Ben Wilson
Lorraine Hewitt