Gene Therapy (All) – Page 15

Stem Cell-Based iNKT Cell Therapy for Cancer

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Post published:June 30, 2025
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Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are…

Process development for establishing an iPSC-based therapeutic candidate for Canavan disease

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Post published:June 30, 2025
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Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This candidate has the potential to…

Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

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Post published:June 30, 2025
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Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs…

Cellular Immune Tolerance Symposium

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Post published:June 30, 2025
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Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

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Post published:June 30, 2025
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Optogenetic Gene Therapy for Treatment of Retinitis Pigmentosa

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Post published:June 30, 2025
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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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Post published:June 30, 2025
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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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Post published:June 30, 2025
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A Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus

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Post published:June 30, 2025
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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…

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

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Post published:June 30, 2025
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Therapeutic Candidate or Device SENTI-202 is an allogeneic off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cellular therapy targeting CD33 and/or FLT3 malignancies. Indication CD33 and/or FLT3 expressing hematologic malignancies,…

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