ProQR Receives Orphan Drug Status for F508del Therapy

[GenH]

ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level.

I have explained this further here:

http://sixtyfiverosesblog.wordpress...eives-orphan-drug-status-for-f508del-therapy/

 

[erock77]

Why is there so little information on this? Even on their website there's little detail. Has it not started any human trial studies yet?

 

[GenH]

Yes I've noticed there is not much information at the moment. Trials will hopefully start next year.

I saw an article about another group looking at a similar RNA approach, they were targetting nonsense mutations. Will post about this when I have the time.