Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With Choroideremia

This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).

This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM.

Key Inclusion Criteria:

• Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
• Both eyes must have ≥ 34 ETDRS letters (~20/200)

Key Exclusion Criteria:

• Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
• Patient has previously received any AAV treatment
• Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)