The Wave 1 gene therapy trial involves the CF gene delivered by liposomes. Trial dosing is likely to finish around June, with results hopefully announced in October.

The Wave 2 product appears to be a more effective form of gene therapy. This involves a CF gene delivered by a virus. Trials will hopefully start early 2017.

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Galapagos are developing new potentiators that have a superior effect on CFTR opening than Kalydeco. The superior opening was seen with class 2, 3 and 4 mutations. It is planned to start a trial by the end of 2014.

New correctors are also being developed in colloboration with AbbVie. It is planned to have a corrector in trials by the end of 2015.

Phase 3 confirmatory trial planned to start early 2014. Conditional EMA application also planned for early 2014.

New graphs show stability in the previous phase 3 trial with those not on inhaled antibiotics. After 48 weeks those taking Ataluren (no antibiotics) dropped 0.2%.

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There are multiple approaches that may help nonsense mutations.

It is thought that Ataluren helps ‘read through,’ allowing the ribosome to ignore the stop codon and produce the normal protein.

A new approach may increase this protein function further, when used in conjunction with Ataluren.

Other Gating: Application submitted to FDA and EMA

R117H: Results expected this year, potential FDA application early 2014

F508del homozygotes: Phase 3 trials underway, data expected mid 2014

F508del heterozygotes: Phase 2 VX809/Kalydeco trial has commenced

A recent study suggested that VX809 helps the F508del mutation by improving several interactions between regions in the CFTR protein.

This study suggested that VX809 interacts with membrane spanding domain one and can help several other CF mutations.

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Novoteris recently announced that the FDA has granted orphan drug designation for inhaled nitric oxide therapy.

The pilot trial demonstrated an 8.8% absolute improvement from baseline. Phase 2 trials are planned for 2014.

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