Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

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Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs…

Continue ReadingOverexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease

A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy…

Continue ReadingA Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease

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

Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

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Therapeutic Candidate or Device Autologous Human CD34+ HSC from Mobilized PBSC of Patients with Cystinosis Modified by Ex Vivo Transduction using the pCCL-CTNS Lentiviral Vector Indication Cystinosis - An autosomal…

Continue ReadingPhase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site: PARADIGM

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Therapeutic Candidate or Device Human pancreatic islets and parathyroid gland combination graft Indication Patients with established Type 1 diabetes Therapeutic Mechanism Pancreatic islet transplantation has become a more viable approach…

Continue ReadingPancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site: PARADIGM

Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus

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Therapeutic Candidate or Device Autologous Ex Vivo Expanded Polyclonal CD4+CD25+CD127lo/-FOXP3+ Regulatory T-cells (CLBS03) Indication Early Onset Type 1 Diabetes Mellitus with Residual Beta Cell Function Therapeutic Mechanism It must be…

Continue ReadingPhase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus