Nightstar Therapeutics Expands Pipeline with Novel Gene Therapy for the Treatment of Stargardt Disease

Nightstar Therapeutics (Nightstar) has recently announced the addition of an exclusive global licence for a gene therapy program for Stargardt disease from Oxford University Innovation.

Nightstar was formed from initial work supported by the Tommy Salisbury Choroideremia Fund at Fight for Sight and is a spin-out company from the University of Oxford.

Stargardt disease (or Stargardt macular dystrophy) is an inherited eye condition that affects predominantly the macula. It’s a rare condition, with prevalence between 1 in 8,000 to 10,000 individuals worldwide. It initially causes blurred vision, but as the disease progresses symptoms can include loss of central vision, problems with colour vision, sensitivity to light and, in some cases, being slow to adapt to the dark.

The University of Oxford developed a novel technology, which is being utilised in the gene therapy programme. The therapy targets the ABCA4 gene, the mutations of which have been linked to Stargardt disease. There are currently no treatment options for this disease.

Nightstar CEO, Dave Fellows, said “The licensing of this novel gene therapy program exemplifies our commitment to developing treatments for patients suffering from inherited retinal diseases that would otherwise lead to blindness. We plan to leverage our clinical experience with choroideremia and X-linked retinitis pigmentosa to accelerate the development of this exciting program for the unmet medical need in Stargardt disease.”

Fight for Sight CEO, Michele Acton, said “We are pleased that Nightstar is accelerating its pipeline of research with the aim of addressing Stargardt through its gene therapy program.”