Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis
Therapeutic Candidate or Device NXC-201 Indication AL Amyloidosis Therapeutic Mechanism Genetically modified T-cell targeting B-cell maturation antigen (BCMA) Unmet Medical Need Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma…
Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients
Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias…
Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant
Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological…
Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant
Therapeutic Candidate or Device COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host Indication Older (>40 yrs) Severe Aplastic Anemia…
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome
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…
LADICell
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…
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
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…
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
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…
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
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…
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