"To date, no pharmacological intervention has been approved for
Primary Ciliary Dyskinesia (PCD) indication"
Live births are affected by PCD
of patients develop bronchiectasis
of PCD patients experience life-threatening respiratory failure and require lung transplantation
approved treatment for PCD
Bring an effective and evidence-based therapeutic solution specific for the treatment of Primary Ciliary Dyskinesia
We are a clinic-ready stage, biotechnology company developing new inhaled therapies for the treatment of a rare genetic disorder, Primary Ciliary Dyskinesia or PCD.
Primary Ciliary Dyskinesia (PCD) is a rare and neglected disease. Patients experience chronic cough, respiratory problems, and recurrent ear and lung infections from birth onwards. Exacerbation of respiratory distress leads to frequent ER visits, hospitalization, poor quality of life for patients, and a huge economic burden. There is currently no pharmacological therapy approved specifically for the treatment of PCD. It is a critical and urgent medical need that must be addressed.
Our lead candidate is specifically designed to address acute symptoms and respiratory distress experienced by PCD patients. In the long term, it will also prevent recurrent infections, impede the lung damage and progression of the disease, and thus improve patients' quality of life.
We plan to further develop our portfolio with novel drug candidates dedicated to the treatment of other rare obstructive airway respiratory diseases such as Cystic Fibrosis (CF). We also plan to explore the therapeutic potential of our drug-candidate to address COVID-19 sequelae.
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.
We have a robust early-stage pipeline with both new chemical entities (NCE) and repurposed candidates