LADICell

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Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Leukocyte Adhesion Deficiency-I (LAD-I). Indication The target clinical indication is Leukocyte Adhesion Deficiency-I (LAD-I), a…

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A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

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Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized…

Continue ReadingA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

  • Post author:
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Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized…

Continue ReadingA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

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Therapeutic Candidate or Device ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia Indication Transfusion-dependent beta-thalassemia Therapeutic Mechanism ST-400 is intended to disrupt BCL11A erythroid enhancer in…

Continue ReadingA Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia

Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant

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Therapeutic Candidate or Device Haploidentical (half-match) T cell depleted blood stem cell transplant with a low-toxic conditioning regimen Indication Adult patients with severe sickle cell disease who are excluded from…

Continue ReadingTreatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant

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

Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)

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Therapeutic Candidate or Device Partially HLA-matched virus-specific T-cell therapy targeting cytomegalovirus, Epstein-Barr virus, and adenovirus. Indication This study will treat persistent viral infections with CMV, EBV, and/or adenovirus in patients…

Continue ReadingAntiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES)

Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning

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Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked…

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Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase - Severe Combined Immunodeficiency (or…

Continue ReadingEfficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects