Manros Therapeutics
From Sea to Pharmacy

Targeted Research Involving Protein Kinases

Our research is focused on protein kinases and their pharmacological inhibitors.

Kinases are responsible for phosphorylation, the addition of a phosphate group to a biological molecule. This reaction is involved in essentially all physiological and cellular events and abnormalities in phosphorylation are observed in many human pathologies, notably proliferation-associated diseases.

 

Cystic Fibrosis (CF)

A rare orphan disease…

CF is a life-threatening and chronically debilitating condition affecting people from very early in the childhood with a frequency of about 1 in 2,500 live births among Caucasians.

  • It affects more than 80.000 patients in the world
  • 33.000 in the USA
  • 40.000 in Europe
  • 6.500 in France.

CF is a genetic disease (autosomal recessive disorder) caused by different mutations of the CF transmembrane conductance regulator (CFTR) gene. There are currently over 2,000 mutations affecting CFTR, many of which give rise to a disease phenotype. The most commonly observed mutation in the world – including the European community – is F508del-CFTR. Indeed, around 75% of CF alleles contain the ΔF508 mutation in which a triplet codon has been lost, leading to the deletion of phenylalanine at position 508 in the protein.

… that affects respiratory functions

Patients suffering from CF develop debilitating lung function disorders. Many patients with CF develop also secondary chronic bacterial pulmonary infections among which Pseudomonas aeruginosa infections are the more frequently reported – 70 to 80% CF patients being primarily infected by their teen years – and, allergic fungal infections such as bronchopulmonary aspergillosis. In the early 2000, 80 to 95% of patients with CF still succumbed to respiratory failure brought on by chronic bacterial infection and concomitant airway inflammation.

Among CF complications, the most frequently reported are cystic fibrosis-related diabetes (CFRD) (20-50% of CF adolescents and adults) and liver (30%) and pancreatic enzyme production (5-10 %) related diseases.