A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

NARecruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

October 30, 2024

Primary Completion Date

October 31, 2026

Study Completion Date

October 31, 2026

Conditions

MECP2 Duplication Syndrome

Interventions

GENETIC

HG204

The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose

Trial Locations (1)

RECRUITING

Peking University First Hospital, Beijing