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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Open-label, Multidose Dose-escalation Study to Understand the Safety of CRISPR Gene-editing Therapy and Its Long-Lasting Effects in DMD Patients (MUSCLE)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingEarly phase 12026-08-03NCT06594094
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderRecruitingEarly phase 12025-08-27NCT07063251
CRISPR/cas13-medIated RNA TarGeting THerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)Neovascular Age-related Macular Degeneration(nAMD)RecruitingEarly phase 12025-03-17NCT06031727
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)syndromic X-linked intellectual disability Lubs typeRecruitingNot applicable2024-11-26NCT06615206
Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trialeye disorder, macular degeneration, retinal degeneration, retinal disorderNot yet recruitingPhase 12024-10-02NCT06623279
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)Eye Diseases, Hereditary, Leber congenital amaurosis, eye disorder, retinal disorderActive, not recruitingEarly phase 12024-09-19NCT06088992