NSR-RPGR, a gene therapy treatment for X-linked retinitis pigmentosa, showed positive preliminary results in a phase 1/2 dose-escalation study, Nightstar Therapeutics announced in a press release.
The open-label, dose-ranging, single-eye XIRIUS trial, which took place in both the United States and United Kingdom, consists of six cohorts of three patients receiving a single subretinal injection. Doses ranged from 5×10^9 genome particles (gp) in the first cohort to 5×10^11 gp in the sixth cohort, the release said.
Study data were released at the Euretina meeting.
All of the patients in