Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy

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Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…

Continue ReadingProviding a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy

Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies

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Research Objective Approximately 20,000 babies are born annually with kidney disease; the long-term outcome is poor. These studies address new ways to develop mini-kidney structures for transplantation to induce repair.…

Continue ReadingEnhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies

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.

Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients

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Therapeutic Candidate or Device Combined hematopoietic stem cell graft and recipient T regulatory cells Indication Kidney disease requiring kidney transplantation Therapeutic Mechanism The study will determine whether patients treated with…

Continue ReadingInduction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients

Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients

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Therapeutic Candidate or Device MDR-101 is cellular therapy consisting of kidney donor-derived CD34+ HSCs and CD3+ T-cells. Indication Maintenance of kidney allograft function after withdrawal of post-transplant immunosuppressant (IS) drugs…

Continue ReadingCellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients

A Phase 3 Study Comparing the Utility of Human Acellular Vessels to Arteriovenous Fistula in Subjects with End-Stage Renal Disease (California Sites)

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Therapeutic Candidate or Device Human Acellular Vessel (HAV) Indication Conduit for Vascular Access for Hemodialysis Therapeutic Mechanism Mechanism of action: the HAV is comprised of intact extracellular matrix constructed by…

Continue ReadingA Phase 3 Study Comparing the Utility of Human Acellular Vessels to Arteriovenous Fistula in Subjects with End-Stage Renal Disease (California Sites)