Phase 2 Gene Therapy Trial Begins Enrollment


A phase 2 clinical trial assessing the safety and efficacy of a gene therapy approach to treatment of choroideremia has begun enrolling patients at the University of Miami Miller School of Medicine.

Researchers will deliver a wild-type copy of the Rab-escort protein 1 (REP1) gene using an adeno-associated virus (AAV2-REP1). The open-label study will enroll six male patients, each of whom will receive a single dose of AAV2-REP1 via subretinal injection.

Choroideremia, an X-linked recessive disease, is caused by mutations to genes that encode REP1. The disease, for which no treatment currently exists, affects 1 in 50 000 people.


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