This conference will bring together clinical and translational investigators in rare genetic causes of bronchiectasis, including primary ciliary dyskinesia (PCD), primary immunodeficiencies (PID), alpha-1 anti-trypsin disease (AATD) and cystic fibrosis (CF), to discuss preparing for collaborative multicenter clinical trials. In this day and a half-long conference presenters will address critical knowledge gaps in disease pathogenesis, clinical trial preparedness and overcoming barriers to successful trials in these rare disease patient populations. Speakers will provide state-of-the-art updates on the diagnosis and major sources of morbidity in these conditions, as well as commonalities and differences in bronchiectasis pathogenesis, with an emphasis on potential therapeutic targets. Lessons learned from previous clinical trials, particularly in CF, will be presented, in order to capitalize on previous experience as we plan future clinical trials. The strengths and weaknesses of potential clinical trial endpoints will be discussed. Innovative study designs for rare diseases and partnering with stakeholders will be highlighted. Junior investigators will be invited to participate in special sessions regarding career development and funding opportunities. Patients and families will have the opportunity to participate in “Meet the Expert” sessions.

Also, on the agenda:

• Mucous Rheology & Mucus Clearance
• Airway Inflammation and Oxidative Stress
• Microbiome and Respiratory Infection
• Dysregulated Immunity (Innate and Adaptive)
• Lung function: Spirometry & Lung Clearance Index

8.75 AMA PRA Category 1 Credits™ – maximum

Venue: Children’s Hospital Colorado