Sep 24, 2021

MeiraGTx and Janssen Recruiting for Phase 3 XLRP Gene Therapy Clinical Trial

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MeiraGTx, a gene therapy development company, and Janssen Pharmaceuticals are recruiting participants for LUMEOS, their Phase 3 gene therapy clinical trial for males and females, three years of age and older, with X-linked retinitis pigmentosa caused by mutations in the gene RPGR. The trial is taking place at several sites in the US and Europe.

MeiraGTx’s gene therapy is designed to deliver functional copies of the RPGR gene to the subretinal space with the goal of improving or preserving vision. The gene therapy is designed to be effective for several years, perhaps the lifetime of the patient.

The subretinal injections deliver copies of a healthy RPGR gene into the patient’s retina to augment the mutated RPGR copies. Human-engineered viral containers — derived from an adeno-associated virus or AAV — deliver the RPGR copies into the photoreceptors, the vision-enabling cells affected by XLRP.

XLRP affects about 20,000 people in the US and EU. As an X-linked condition, XLRP primarily affects males, although some females may be affected, sometimes significantly.