A gene therapy gives children losing their sight their first real shot at driving a car

Durham, North Carolina's Atsena Therapeutics reported on October 7 that its gene therapy ATSN-201 for X-linked retinoschisis — a disease with no approved treatments — showed clinically meaningful vision gains in its LIGHTHOUSE study. Three of the nine patients treated at the pivotal dose reached 20/40 vision or better, the threshold for driving eligibility in the US, with improvements building over time. Enrollment in the pivotal Phase 3 portion is now progressing rapidly, a sign of how urgently doctors and patients are waiting for a treatment.