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🫁PULMONOLOGY

Lung Disease

COPD, cystic fibrosis, pulmonary fibrosis β€” breathing easy through gene correction and regeneration

~545 million people live with chronic respiratory disease worldwide; COPD alone is the 3rd leading cause of death.
CURE AVAILABLE
50/ 100
TO BROAD CURE

MILESTONETrikafta is FDA-approved for cystic fibrosis; Jascayd is EU-approved for IPF.

LATESTJideytro got US FDA approval for ROS1-positive NSCLC.

An approved cure exists for a major form of this disease Β· Not medical advice.

CURRENT STATUS

The field has split into a near-cure and a set of hard frontiers. In cystic fibrosis, CFTR modulators (elexacaftor/tezacaftor/ivacaftor) restore protein function in ~90% of patients and have converted a fatal pediatric disease into a manageable adult one; inhaled mRNA and gene therapy target the remaining ~10% with non-modulatable mutations. In COPD the first biologic (dupilumab) is now approved for type-2 inflammation, while true alveolar regeneration remains preclinical. In idiopathic pulmonary fibrosis, next-generation antifibrotics (PDE4B inhibition) are in Phase 3 aiming to move beyond slowing toward stabilization.

KEY BREAKTHROUGHS

Trikafta β€” CFTR modulator restoring ~90% of lung function in eligible CF patients

Inhaled mRNA therapy delivering CFTR to airway cells in Phase I trials

AI identifying novel anti-fibrotic targets for IPF from single-cell lung atlases

CRISPR correction of Ξ”F508 CFTR mutation in patient-derived organoids

AI-COMPRESSED PIPELINE

AI TOOLS ACCELERATING CURES

Lung Imaging AIInhaled mRNA DesignCRISPR Airway DeliveryFibrosis Prediction Models

KEY ORGANIZATIONS

Vertex PharmaceuticalsArctus TherapeuticsFlagship PioneeringBoehringer IngelheimCFF

KEY CLINICAL TRIALS

FIBRONEER-IPF β€” Nerandomilast for Idiopathic Pulmonary Fibrosis (Phase 3)

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Boehringer Ingelheim

An oral PDE4B inhibitor that met its Phase 3 primary endpoint, significantly slowing the decline in lung function (FVC) over 52 weeks β€” the first major IPF advance since the antifibrotics of the 2010s, with both antifibrotic and anti-inflammatory effects.

πŸ‘₯ ~1,177 participants with idiopathic pulmonary fibrosisπŸ“ Global β€” multicenter

FIBRONEER-ILD β€” Nerandomilast for Progressive Pulmonary Fibrosis (Phase 3)

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Boehringer Ingelheim

Companion trial extending nerandomilast to progressive fibrosing interstitial lung disease beyond IPF. Also met its primary endpoint, slowing FVC decline versus placebo β€” broadening the reach of the first new antifibrotic mechanism in a decade.

πŸ‘₯ ~1,176 participants with progressive pulmonary fibrosisπŸ“ Global β€” multicenter

TIMELINE ESTIMATE

Cystic fibrosis gene cure: 3–5 years. Pulmonary fibrosis disease-modifiers: 4–6 years. COPD regeneration: 6–10 years.

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