A clinic-ready stage, biotechnology company developing new inhaled therapies for the treatment of a rare genetic disorder, Primary Ciliary Dyskinesia(PCD), and other respiratory diseases.
"To date, no pharmacological intervention has been approved for
Primary Ciliary Dyskinesia (PCD) indication"
Live births are affected by PCD
PCD patients experience life-threatening respiratory failure and some require lung transplant
Drugs approved for PCD indication
Help people with airway obstructive diseases breathe easily and prevent progressive lung damage & respiratory failure
What is Primary Ciliary Dyskinesia (PCD)?
PCD is a rare disease caused by multiple genetic mutations involving fingerlike projections on airway cells known as "Cilia". It is estimated to affect ~1/10000 birth in the US.
Normal mucus traps pathogens and Cilia move to clear it. In PCD patients, due to genetic reasons, Cilia are dysfunctional so mucus builds up and forms "mucus plugs". These plugs clog airways and initiate a vicious cycle of recurrent infection, airway damage, permanent lung damage, and progression to bronchiectasis.
Source: NHLBI website
Source: Mirra et al., 2017. Frontiers in Pediatrics.
Patients experience chronic cough, respiratory problems, and recurrent ear and lung infections from birth onwards. A significant number of PCD cases experience life-threatening respiratory failure and some require lung transplantation. Exacerbation of respiratory distress leads to frequent ER visits, hospitalization, poor quality of life for patients, and a huge economic burden on families and the healthcare system.
CILA has a robust early-stage pipeline