Cystic Fibrosis

Disease overview
Cystic Fibrosis is a recessive chronic progressive genetic disorder of the lung with high morbidity and shortened life expectancy. The condition occurs as a consequence of differing hereditary mutations in the gene encoding for the CFTR protein, leading to mucus accumulation. This results in airway obstruction, chronic lung infection and bronchiectasis, which in aggregate causes progressive deterioration in the patient, until poor ventilation of the lung ultimately leads to heart failure. The mean age of death is 40 and the median age is 27. Cystic Fibrosis affects approximately 70,000 patients worldwide.

Evidence base
LAMELLASOME™ CF-NA is genotype-agnostic treatment that corrects the basic defect in the CFTR chloride ion channel which causes Cystic Fibrosis. It is intended that LAMELLASOME™ CF-NA will be administered monthly by nebulisation. 

   Characteristic  Existing evidence  Type
EFFICACY LAMELLASOME™ CF-NA efficacy Lamellar and the Roslin Institute have shown that LAMELLASOME™ CF-NA is uniquely effective in a validated model.


SAFETY LAMELLASOME™ respiratory safety Non-clinical respiratory safety program on LAMELLASOME™ completed demonstrating an excellent safety and tolerability profile.


  LAMELLASOME™ CF-NA individual components safety The individual components of CF-NA have been shown to be safe and well tolerated in human studies. Clinical testing


Competitive edge
Cystic Fibrosis is caused by mutation of the CFTR gene of which there are believed to be around 2,000 types. LAMELLASOME™ CF-NA addresses this basic defect in the CFTR chloride ion channel irrespective of the mutation genotype. Existing CFTR modifying medications tend to be genotype specific treating only a proportion of the Cystic Fibrosis population. Additionally, some existing treatments have notable side-effects whereas LAMELLASOME™ has been proven to be safe and well tolerated in the lung to date. LAMELLASOME™ CF-NA promises to provide a safer and more effective treatment.

Development route
Cystic Fibrosis qualifies as an orphan disease indication in both the EU & US. This is anticipated to provide the benefit of an accelerated development program and expedited regulatory review.

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