Disease Focus: Metabolic Disorders

Personalized Cell Therapy for Diabetes

Translational Candidate Preclinical studies will develop patient specific stem cell-derived islets that secrete insulin & other islet hormones for regulation of blood sugar Area of Impact Genetically matched stem cell derived islets could provide treatment for diabetes without the need for immunosuppression or implantable devices. Mechanism of Action The stem cell-derived islets contain insulin-producing pancreatic […]

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

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 and Sandhoff disease patients who have no curative or ameliorating treatment. Mechanism of Action Wild type HexA and HexB will be delivered to affected neurons […]

ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy

Translational Candidate ASCENT – Advanced Superdonor Cellular Enteric Neuropathy Therapy, is a donor progenitor cell population that replaces the enteric nervous system. Area of Impact ASCENT would treat enteric neuropathies including Hirschsprung disease and total intestinal aganglionosis which currently have no direct therapy Mechanism of Action Our goal is to develop an allogeneic “off the […]

Cellular Immune Tolerance Symposium

JDRF Encapsulation Consortium Fall 2017 Meeting

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

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 Mucopolysaccharidosis I (MPSI) is a rare disease that affects predominantly children. Untreated, these patients typically die by the age of 10. Therapeutic Mechanism The therapeutic […]

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

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 metabolic disease that belongs to the family of the lysosomal storage disorders. Gene involved is CTNS (encodes cystinosin). Therapeutic Mechanism The proposed therapy intervention is […]

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

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 to treat patients with established Type 1 diabetes. However, widespread application has been limited by several barriers, most importantly, poor islet survival and an inability […]

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

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 acknowledged that the mechanism(s) by which the effector arm of the immune system becomes unrestrained in the setting of T1D, resulting in the immune destruction […]

Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes

Therapeutic Candidate or Device pancreatic progenitor cells in a delivery device that allows direct vascularization Indication high-risk type 1 diabetes including "brittle" diabetes and hypoglycemia unawareness Therapeutic Mechanism People with type 1 diabetes have lost their pancreatic cells that make insulin, and therefore have to self-administer insulin. It is very difficult to manage blood sugar […]