An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency Post author: Post published:April 20, 2026 Post category: Continue ReadingAn Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector Post author: Post published:April 20, 2026 Post category: Continue ReadingGene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Post author: Post published:April 20, 2026 Post category: Continue ReadingStudy of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
CCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer Post author: Post published:April 20, 2026 Post category: Continue ReadingCCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease Post author: Post published:April 20, 2026 Post category: Continue ReadingA Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy)) Post author: Post published:April 20, 2026 Post category: Continue ReadingStudy of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy))
Gene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia Post author: Post published:April 20, 2026 Post category: Continue ReadingGene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) Post author: Post published:April 20, 2026 Post category: Continue ReadingA Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Post author: Post published:April 20, 2026 Post category: Continue ReadingSafety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) Post author: Post published:April 20, 2026 Post category: Continue ReadingPhase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
