Phase I/II Clinical Trial for CMT4J

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Clinical Development of ETX101, a Gene Regulation Therapy for SCN1A+ Dravet Syndrome

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

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

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

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Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa

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Phase 1b Study of TN-401 in Adults with PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

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TRX103 for prevention of GvHD in patients receiving HLA mismatched HSCT for the treatment of hematologic malignancies

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Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS-ADA lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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

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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…

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