Adult or Tissue Stem Cell – Page 13

Stem Cell-Based iNKT Cell Therapy for Cancer

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Post published:June 4, 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…

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

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Post published:June 4, 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…

Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome

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Post published:June 4, 2025
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A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1)

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Post published:June 4, 2025
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Therapeutic Candidate or Device Cryopreserved autologous TRAC locus 1XX BCMA-CAR T cells Indication Relapsed and Refractory Multiple Myeloma Therapeutic Mechanism Engineered T cells target and kill BCMA+ Myeloma cells Unmet…

Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors

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Post published:June 4, 2025
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Therapeutic Candidate or Device Engineered CAR T-cells are enhanced to allow their survival in solid tumors Indication Children, adolescents and young adults with variety of solid tumors but focus on…

Extracellular Vesicles for Ventricular Tachycardia

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Post published:June 4, 2025
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Therapeutic Candidate or Device CSSHI-2003 (extracellular vesibles derived from cardiohsphere derived cells) Indication Patients afflicted with recurrent fast and irregular heart beating (a.k.a. ventricular tachycardia). Therapeutic Mechanism CSHHI-2003 (CDC exosomes)…

Neural stem cell delivered CRAd-S-pk7 oncolytic viro-immunotherapy for ovarian cancer

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Post published:June 4, 2025
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Therapeutic Candidate or Device A clinically tested tumor-tropic neural stem cell (NSC) platform for effective distribution of oncolytic virotherapy to ovarian cancer metastases. Indication Chemo-resistant, metastatic ovarian cancer. Therapeutic Mechanism…

Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease

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Post published:June 4, 2025
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Therapeutic Candidate or Device Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis. Indication The proposed studies provide an innovative stem cell based approach with gene…

Superior forward-oriented b-globin vector for treating Sickle Cell Disease

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Post published:June 4, 2025
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Therapeutic Candidate or Device forward-oriented globin-expressing vector CD34+ HSCs Indication Severe Sickle Cell Disease (SCD) Therapeutic Mechanism Successful gene modified HSC generate RBC with effective repaired hemoglobin in 30% of…

Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia

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Post published:June 4, 2025
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Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing…

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