DISAPPOINTING NEWS IN THE GENE THERAPY WORLD

Last year, we wrote an article on the use of Gene Therapy in ophthalmology which was published in The Ophthalmologist (link in comments). One company we featured was ProQR Therapeutics who are developing RNA therapies for inherited retinal diseases. ProQR recently announced their TopLine Phase 2/3 results from their lead program (Sepofarsen) which unfortunately came back with some disappointing results.

The ‘study did not meet primary endpoint nor notable secondary endpoints’. “Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10” Daniel A. de Boer, Founder and CEO of ProQR Therapeutics.

Although this is disappointing news, what they and the other companies we included in our Gene Therapy article are attempting to do is to cure blindness for certain diseases. This has always been a huge challenge and companies shouldn’t be discouraged by negative clinical trail results as they usually have multiple programmes that they can apply their new findings to.

The only way we can make progress on these awful diseases is by trying our best and failing, but we should learn from what hasn’t worked. 

Ref: ProQR Announces Top-Line Results from Phase 2/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10 | ProQR Therapeutics