Disease Focus: Metabolic Disorders
Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting
Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene for & produce […]
Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy
Translational Candidate 'Pseudoislets' derive from human islets, but compared to islets have superior survival, function, and diabetes reversal after transplantation. Area of Impact Pseudoislets could transform islet replacement strategies in diabetes by increasing the number and durable function of transplanted islet cells. Mechanism of Action Transplantation of replacement human islet cells is approved in type […]
MPS II: Plasma cell delivery of iduronate sulfatase
Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II, a rare genetic disease causing multi-organ symptoms and death by age 15, if not treated. Current treatment does not address major symptoms. Mechanism of Action […]
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 […]
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 […]