Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

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Therapeutic Candidate or Device Cytomegalovirus (CMV)-specific T cells that express a chimeric antigen receptor (CAR) which targets and eliminates HIV-infected cells Indication Management of human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome…

Continue ReadingEvaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

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Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication…

Continue ReadingA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

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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…

Continue ReadingReduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205

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Therapeutic Candidate or Device AB-205 is an intravenously infused medicine containing genetically modified human vascular (endothelial) cells intended to repair damaged tissue Indication Treatment to reduce/prevent severe multi-organ complications from…

Continue ReadingAB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

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Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic…

Continue ReadingA Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant

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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…

Continue ReadingSequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant

Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant

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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…

Continue ReadingTreatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant