Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects

The primary objective of this study is to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer to healthy participants and patients with Primary Ciliary Dyskinesia caused by a pathogenic mutation in the DNAI1 Gene.

Major Inclusion Criteria:

• Healthy, adult, male or female of, 18-75 years of age, inclusive, at screening.
• Participant has disease causing mutations in the DNAI1 gene
• The participant has a forced expiratory volume in one second (FEV1) of at least 50% predicted.

Major Exclusion Criteria:

• History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
• History of cancer, with exception of adequately treated basal cell or squamous cell carcinoma of the skin.
• Medically significant hemoptysis
• Anticoagulation therapy for the treatment of a pulmonary embolus or has had a pulmonary embolus in the last 6 months of screening.
• Active tuberculosis infection.
• Laboratory abnormalities in clinical laboratory tests at screening:

• Serum creatinine level
• Total bilirubin, aspartate aminotransferase or alanine aminotransferase values
• Hematological or coagulation values outside the normal reference range
• Any medical history of disease that has the potential to cause a rise in total bilirubin over the ULN
• History of alcohol abuse or drug addiction with the last year of screening.
• Active smoker (vaping included).

Other protocol defined inclusion/exclusion criteria may apply.