Making Connections: Nightstar Therapeutics

I was so happy to recently speak with Samantha Vieira, Senior Director of Program Management of Nightstar Therapeutics. Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel, one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness.

Based in London, they also have a small office in Lexington, Mass., and have several projects focused on retinal disease in their pipeline.   

Their mission is to maintain and restore sight in patients with inherited retinal diseases and they shared information about four projects currently in their pipeline to treat these inherited retinal diseases: Choroideremia, X-linked Retinitis Pigmentosa (XLRP), Best Vitelliform Macular Dystrophy (MD) and Stargardt disease.  

Choroideremia a degenerative, X-linked genetic retinal disorder primarily affecting males is expected to enter stage 3 clinical trials in early 2018.  Very exciting news!  

Their website includes helpful overviews of the four diseases for which they have projects, and some great videos as well.  If you are interested in learning more about their research or upcoming clinical trials, there is also a sign up form for patients and families here.  

As always, I’m encouraged to meet a company that is dedicated to finding treatments for rare inherited retinal diseases, and I look forward to hearing more about their work in 2018!