Advancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing Post author: Post published:April 21, 2026 Post category: Continue ReadingAdvancing ISP-001 for Mucopolysaccharidosis I: Clinical Expansion, Clinical Manufacturing and Release Testing
Autologous iPSC-derived Retinal Pigment Epithelium Cell Therapy to Restore Vision in Blinding Eye Disease Post author: Post published:April 21, 2026 Post category: Continue ReadingAutologous iPSC-derived Retinal Pigment Epithelium Cell Therapy to Restore Vision in Blinding Eye Disease
A Phase 1 trial of RNDP-001, a dopaminergic progenitor drug product, in idiopathic Parkinson’s Disease Post author: Post published:April 21, 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:April 21, 2026 Post category: Continue ReadingClinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome
Phase I/II Clinical Trial for CMT4J Post author: Post published:April 21, 2026 Post category: Continue ReadingPhase I/II Clinical Trial for CMT4J
Phase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD) Post author: Post published:April 21, 2026 Post category: Continue ReadingPhase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD)
Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa Post author: Post published:April 21, 2026 Post category: Continue ReadingOptogenetic Gene Therapy for Treatment of Retinitis Pigmentosa
TRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies Post author: Post published:April 21, 2026 Post category: Continue ReadingTRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies
Inhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy Post author: Post published:April 21, 2026 Post category: Continue ReadingInhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy
Phase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) Post author: Post published:April 21, 2026 Post category: Continue ReadingPhase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
