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

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The UCLA Delayed Immunological Tolerance after Kidney Transplantation Program

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Selective, Off-the-Shelf Logic Gated CAR NK Cell Therapy Targeting CD33 and/or FLT3 Expressing Hematologic Malignancies

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Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis

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RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration

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A PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)

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A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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