Cellular Immune Tolerance Symposium Post author: Post published:May 23, 2026 Post category: Continue ReadingCellular Immune Tolerance Symposium
Advancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing Post author: Post published:May 23, 2026 Post category: Continue ReadingAdvancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing
Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, a gene therapy for Pitt Hopkins Syndrome Post author: Post published:May 23, 2026 Post category: Continue ReadingPhase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, a gene therapy for Pitt Hopkins Syndrome
Phase I/II Clinical Trial for CMT4J Post author: Post published:May 23, 2026 Post category: Continue ReadingPhase I/II Clinical Trial for CMT4J
Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome Post author: Post published:May 23, 2026 Post category: Continue ReadingClinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome
Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector Post author: Post published:May 23, 2026 Post category: Continue ReadingGene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector
Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa Post author: Post published:May 23, 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:May 23, 2026 Post category: Continue ReadingPhase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
TRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies Post author: Post published:May 23, 2026 Post category: Continue ReadingTRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies
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:May 23, 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
