Phase I/II Clinical Trial for CMT4J Post author: Post published:March 30, 2026 Post category: Continue ReadingPhase I/II Clinical Trial for CMT4J
A Phase 1 trial of RNDP-001, a dopaminergic progenitor drug product, in idiopathic Parkinson’s Disease Post author: Post published:March 30, 2026 Post category: Continue ReadingA Phase 1 trial of RNDP-001, a dopaminergic progenitor drug product, in idiopathic Parkinson’s Disease
Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome Post author: Post published:March 30, 2026 Post category: Continue ReadingClinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome
Phase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD) Post author: Post published:March 30, 2026 Post category: Continue ReadingPhase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD)
Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector Post author: Post published:March 30, 2026 Post category: Continue ReadingGene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector
Inhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy Post author: Post published:March 30, 2026 Post category: Continue ReadingInhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy
Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa Post author: Post published:March 30, 2026 Post category: Continue ReadingOptogenetic Gene Therapy for Treatment of Retinitis Pigmentosa
Phase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) Post author: Post published:March 30, 2026 Post category: Continue ReadingPhase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects Post author: Post published:March 30, 2026 Post category: Continue ReadingEfficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects
TRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies Post author: Post published:March 30, 2026 Post category: Continue ReadingTRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies
