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…

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A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

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Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of…

Continue ReadingA Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.

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Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and…

Continue ReadingTRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.

IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome

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Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy…

Continue ReadingIND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome

Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)

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Therapeutic Candidate or Device Blood-forming stem cells harboring a SCID gene defect, modified to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene. Indication Treatment…

Continue ReadingEx Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)