Inhaled gene therapy aims to fix cystic fibrosis lungs
NCT ID NCT05248230
First seen Jun 25, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This phase 1/2 trial tests a single inhaled dose of gene therapy called 4D-710 in 30 adults with cystic fibrosis who cannot take standard modulator drugs. The therapy delivers a working copy of the CFTR gene directly to lung cells. The main goal is to check safety and side effects, but researchers will also look for signs that the treatment is improving lung function.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- 4D-710 (gene therapy delivered via inhalation)
- What this could lead to
- If successful, this could provide a new treatment option for cystic fibrosis lung disease, especially for people who cannot take current modulator therapies.
- What could go wrong
- This is an early phase trial with only 30 participants, so safety and effectiveness are not yet proven. Gene therapy may not reach enough lung cells or could cause side effects.
Why investors are watching
4D Molecular Therapeutics is testing 4D-710, a gene therapy for adults with cystic fibrosis, in a small 30-patient Phase 1/2 trial. For a small-cap company, this readout matters because a single drug's early data can drive its entire valuation and future pipeline decisions.
If it works: A positive result could show the therapy safely delivers the gene and improves disease control, which might support further development and partnerships. Success in this early trial could position 4D-710 as a viable treatment option for a broader cystic fibrosis population.
If it fails: Gene therapies often fail in early trials due to safety issues or lack of effectiveness, and this small study may not prove the drug works. A failure or delay could hurt the company's prospects, as it has few other public catalysts to fall back on.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2022
- Expected to finish
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Jun 2031
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 clinical manifestations of CF lung disease 3. Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects. 3. Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening 4. Resting oxygen saturation ≥ 92% on room air at Screening Key Inclusion Criteria (Sub-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 clinical manifestations of CF lung disease 3. Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period 4. FEV1 ≥ 40% and \< 70% predicted (per Global Lung Function Initiative) at Screening, AND/OR experienced at least 2 pulmonary exacerbations in the last year requiring intravenous antibiotics Key Exclusion Criteria (Primary and Sub Study): 1. Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary) 2. Active Mycobacterium abscessus infection requiring ongoing treatment at Screening 3. Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy 4. Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening; must agree to not smoke or use vaping products during Screening and initial 24-month observation period. 5. Contraindication to systemic corticosteroid therapy 6. Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition 7. If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening 8. If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C \> 7.5% at Screening 9. Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment 10. Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia 11. Body Mass Index (BMI) \< 16 12. Laboratory abnormalities at screening: * ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN) * Total bilirubin ≥ 2 × ULN * Hemoglobin \< 10 g/dL 13. Requirement for continuous or night-time oxygen supplementation 14. Known CF liver disease with evidence of multilobular cirrhosis 15. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
18 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Johns Hopkins Hospital
RECRUITINGBaltimore, Maryland, 21287, United States
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Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
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Medical University of South Carolina
RECRUITINGCharleston, South Carolina, 29425, United States
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National Jewish Health
RECRUITINGDenver, Colorado, 80206, United States
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Northwestern Memorial Hospital
RECRUITINGChicago, Illinois, 60611, United States
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Penn State Health
WITHDRAWNHershey, Pennsylvania, 17033, United States
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Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
RECRUITINGCleveland, Ohio, 44146, United States
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The Hospital of the University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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The University of Arizona
RECRUITINGTucson, Arizona, 85724, United States
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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University of Alabama Child Health Research Unit
RECRUITINGBirmingham, Alabama, 35233, United States
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University of California San Francisco
RECRUITINGSan Francisco, California, 94143, United States
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Miami Hospital
RECRUITINGMiami, Florida, 33136, United States
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University of North Carolina at Chapel Hill
COMPLETEDChapel Hill, North Carolina, 27599, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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Virginia Commonwealth University Health System
RECRUITINGRichmond, Virginia, 23298, United States
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