Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells

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Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children…

Continue ReadingGene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells

A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC

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Therapeutic Candidate or Device Combination therapy with adenoviral CCL21 gene-modified DC and pembrolizumab Indication Patients with confirmed and measurable stage IV NSCLC expressing PD-L1 in less than 50% of cells…

Continue ReadingA phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC

Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma

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Therapeutic Candidate or Device Genetically engineered, Centyrin-based, stem cell memory CAR-T cells (CARTyrin T cells) Indication Multiple Myeloma Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that…

Continue ReadingClinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma

Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

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Therapeutic Candidate or Device KA34 is an intra-articularly delivered small molecule therapeutic candidate which directs the differentiation of endogenous stem and progenitor cells Indication Osteoarthritis Therapeutic Mechanism KA34 promotes the…

Continue ReadingEvaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

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Therapeutic Candidate or Device A promising immunotherapy utilizing a patient’s memory T cells engineered to express chimeric antigen receptors for targeted tumor killing. Indication Malignant glioma (WHO III and IV),…

Continue ReadingPhase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma

Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome

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Therapeutic Candidate or Device Hu5F9-G4 is a drug called an antibody that is designed to mobilize the body's immune system to eliminate cancer and cancer stem cells. Indication Patients with…

Continue ReadingPhase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome

Induction of Tolerance to Combined Kidney and Hematopoietic Progenitor Cell Transplants from HLA Haplotype Matched Living Donors

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Therapeutic Candidate or Device blood stem cells and T cells from organ transplant donors will be studied under this proposal to prevent rejection of kidney transplants Indication to withdraw immunosuppressant…

Continue ReadingInduction of Tolerance to Combined Kidney and Hematopoietic Progenitor Cell Transplants from HLA Haplotype Matched Living Donors

In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major

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Therapeutic Candidate or Device Maternal bone marrow-derived CD34+ hematopoietic stem cells. Indication Fetal alpha thalassemia major. Therapeutic Mechanism This strategy that takes advantage of existing tolerance between the mother and…

Continue ReadingIn Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major

A Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Sub…

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The HIV-1 virus enters cells by binding to a protein called CCR5 on the cell surface. A naturally occurring mutation in CCR5, CCR5d32, has been shown to provide protection from…

Continue ReadingA Phase I, Open-Label Study To Assess The Safety, Feasibility and Engraftment of Zinc Finger Nucleases (ZFN) CCR5 Modified Autologous CD34+ Hematopoietic Stem/Progenitor Cells (SB-728MR-HSPC) with Escalating Doses of Busulfan In HIV-1 (R5) Infected Sub…