Phase I/II Clinical Trial for CMT4J

• Post author:
• Post published: November 9, 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: November 9, 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: November 9, 2026
• Post category:

Continue ReadingPhase 1B/2A study of the safety and tolerability of human neural stem cells for Huntington’s Disease (REGEN4HD)

Inhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy

• Post author:
• Post published: November 9, 2026
• Post category:

Continue ReadingInhibitory Interneuron Cell Therapy (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Epilepsy

Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

• Post author:
• Post published: November 9, 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: November 9, 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: November 9, 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: November 9, 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: November 9, 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

A Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus

• Post author:
• Post published: November 9, 2026
• Post category:

Therapeutic Candidate or Device iPSC-derived CD19 CAR T cell therapy Indication Systemic Lupus Erythematosus Therapeutic Mechanism Depletion of CD19+ B cells and plasmablasts Unmet Medical Need Potential for drug-free remission…

Continue ReadingA Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus