HOW DO PULMONARY GENE THERAPIES WORK?

Pulmonary gene therapies are innovative treatments designed to deliver genetic material, such as DNA, mRNA, small RNAs, or gene-editing components, directly into lung cells to correct, replace, regulate, or silence specific genes. The goal is to restore or modify cellular function in genetic or acquired respiratory diseases and certain lung cancers, while minimizing off-target effects.

The first FDA-approved gene therapy for a respiratory condition, zopapogene imadenovec-drba (Papzimeos), was approved in August 2025 for adults with recurrent respiratory papillomatosis (RRP), a rare HPV-induced disorder that causes benign airway tumors. It is a non-replicating adenoviral vector–based immunogene therapy that delivers HPV types 6 and 11 antigen genes to stimulate an immune response against infected airway cells, helping reduce papilloma growth and recurrence.

Pulmonary gene therapy delivers genetic material to the lungs or respiratory tract. Papzimeos is administered as subcutaneous injections for adults with recurrent respiratory papillomatosis (RRP) to reduce papilloma growth. Other pulmonary gene therapies are under investigation for cystic fibrosis, α-1 antitrypsin deficiency, pulmonary fibrosis, and lung cancer, with potential administration via subcutaneous, inhalation, intrabronchial, pleural, or systemic routes.

Common side effects of pulmonary gene therapy include the following:

• Injection site reactions 
• Nausea
• Fatigue 
• Chills
• Fever 
• Muscle pain

Information contained herein is not intended to cover all possible side effects, precautions, warnings, drug interactions, allergic reactions, or adverse effects. Check with your doctor or pharmacist to make sure these drugs do not cause any harm when you take them along with other medicines. Never stop taking your medication and never change your dose or frequency without consulting your doctor.

Generic and brand names of FDA-approved pulmonary gene therapy include:

• Papzimeos
• zopapogene imadenovec
• zopapogene imadenovec-drba