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 Cystic Fibrosis Transmembrane Conductance Regulator (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.

Muco-ease is designed to maintain mucociliary clearance and slow disease progression in CF from early stages of the disease.

Muco-ease is a LAMELLASOME™ therapeutic currently in development to treat thick CF mucus by reducing its viscoelastic properties, limiting tissue damage and reducing the burden and/or risk of infection. The combined properties of Muco-ease have the potential to slow disease progression in all stages of CF:

  • Enhances mucociliary clearance by working on all the viscoelastic components of CF mucus
  • The activity against mucin, in particular, makes it suitable for intervention early in disease
  • Maintaining mucin fluidity may modify the course of the disease
  • Muco-ease significantly increases the fluidity of DNA compared to the current market leading mucolytic, Pulmozyme which acts only on DNA
  • Reduces the lung’s fibrotic response to inflammation
  • Facilitates microbial clearance by disrupting quorum. Sensing and inhibiting biofilm. Supporting the lung’s immune defence
  • When used with suboptimal dosing of Tazocin IV antibiotic, it provided resolution of lung infection, possibly through antibiotic potentiation and increased mucociliary clearance1

    1Model of PA (LESB65) respiratory infection2. CFU counts following no treatment (PA only), LMS (LAMELLASOME™) alone, Tazocin alone or Tazocin + LMS (*=p<0.05, **=p<0.001)). Treatments administered at 24 and 48 hours post infection.
    2Nat Commun. 2014 Sep 2;5:4780. doi: 10.1038/ncomms5780

Intended as an add-on therapy to existing treatments and to be inhaled via nebulisation once to twice daily, Muco-ease has Orphan Drug Designation in the EU (EU/3/11/896) indicated for the “Treatment of cystic fibrosis”.

Muco-ease is ready to move into the clinic.

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