On Nightstar's ocular gene therapy

Nightstar Therapeutics, a U.K.-based biotech, just brought its experimental gene therapy for X-linked retinitis pigmentosa stateside — introducing new genes to a 23-year-old Puerto Rican man in Miami to help reverse his inherited blindness. Julio Adomo Nieves is the 16th person to join the worldwide trial. The company this weekend releasedproof-of-concept data showing dose-related improvements for the drug.

Retinitis pigmentosa is a popular candidate for gene therapy — Horama, a French biotech, is working on a similar approach to the disease. It makes sense: Spark Therapeutics’ Luxturna, the gene therapy for another inherited eye condition, was approved late last year — costing $425,000 per eye. The patient population there is less than 2,000. Retinitis pigmentosa offers up a larger market — with an estimated 17,000 patients in need of therapy in the U.S. and Europe.