Gene therapy for inherited retinal dystrophies
Fully diluted ownership
Value of Syncona holding
% of nav
Nightstar is a biopharmaceutical company focused on the development and commercialisation of therapies for retinal dystrophies.
Nightstar’s most advanced program, NSR AAV2-REP1, is in clinical trials for the treatment of choroideremia, a rare disease which causes permanent loss of eyesight and for which there is no other treatment. The gene therapy is delivered by injection into the retina, providing a working copy of the disease-causing faulty gene locally in the eye. The disease modifying technology has the potential to maintain and restore sight in patients, from a single administration.
There are multiple inherited forms of blindness that are addressable with Nightstar’s gene therapy technology. Nightstar is building a pipeline of products based on its manufacturing, gene therapy and retinal surgery capability.
- Chris Hollowood
- Iraj Ali
- Robert MacLaren