The Sanofi Genzyme Therapeutic Program for Cystic Fibrosis

[vc_row][vc_column][vc_column_text]Cystic Fibrosis Foundation Therapeutics (CFFT) — the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation — announced a $14 million expansion of its research collaboration with Sanofi Genzyme  in 2015 that aims to develop new cystic fibrosis (CF) therapies.

Sanofi Genzyme, the specialty care global business unit of Sanofi that focuses on rare diseases, is conducting early stage research into several genetic diseases, including cystic fibrosis (CF). The collaboration between CFFT and Sanofi Genzyme began in 2011 with the goal of identifying new correctors for the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF patients with the most common mutation, the F508del mutation. CFFT and Sanofi Genzyme hope the collaboration accelerates the discovery of therapies for these people.

The Sanofi Genzyme CF therapeutic program

CFTR proteins normally serve as channels to allow the transport of water and charged ions, such as chloride, in and out of cells, creating a thin mucus that protects and lubricates internal organs (the lungs and pancreas, for example).

However, if a mutation occurs in the gene that encodes for the CFTR protein, the transport of fluids in cells is compromised. In the F508del mutation, an amino acid is deleted in the 508 position. This causes the CFTR protein to misfold and not move to its proper place on the cell surface, resulting in the production of thick mucus that accumulates in airways. Bacteria and other microbes and foreign particles get trapped in the mucus, leading to repeat cycles of infection and inflammation in CF patients that can become life threatening.

The Sanofi Genzyme CF therapeutic program is still in its preclinical stage, but several correctors have already been identified and shown an ability to position the defective CFTR protein in its proper place on the cell surface. This expansion program aims to further develop some of the correctors identified for F508del mutation and advance them into clinical trials.

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