Project Objective: Clinical Trial, Phase 1 or Phase 1/2
Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning
Therapeutic Candidate or Device Bone marrow stem cells will be transduced with a lentiviral vector to deliver a normal copy of the gamma-chain gene to treat X-linked SCID. Indication x-linked severe combined immunodeficiency, a severe pediatric disorder in which children have multiple defects in their immune system. Therapeutic Mechanism The gene therapy will correct the […]
Pulmonary Arterial Hypertension Treated with Cardiosphere-Derived Allogeneic Stem Cells
Therapeutic Candidate or Device CAP-1002 Indication Patients with Pulmonary Arterial Hypertension (PAH) Therapeutic Mechanism CAP-1002's mechanism of action may result in reduced wall thickening of small blood vessels in the lung that are markedly narrowed/obstructed in PAH. The latter results in high resistance against which the right ventricle (RV) must pump in order to drive […]
Induction of Tolerance to Combined Kidney and Hematopoietic Progenitor Cell Transplants from HLA Haplotype Matched Living Donors
Therapeutic Candidate or Device blood stem cells and T cells from organ transplant donors will be studied under this proposal to prevent rejection of kidney transplants Indication to withdraw immunosuppressant drugs from kidney transplant recipients Therapeutic Mechanism Injection of the donor blood stem cells into recipients will prevent recipient immune cells from rejecting the donor […]
Human Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this factor to dying motor neurons. Unmet Medical Need There is no treatment […]
In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major
Therapeutic Candidate or Device Maternal bone marrow-derived CD34+ hematopoietic stem cells. Indication Fetal alpha thalassemia major. Therapeutic Mechanism This strategy that takes advantage of existing tolerance between the mother and fetus during pregnancy, so that maternal cells can be transplanted into a fetus without conditioning or immunosuppression. Survivors of alpha thalassemia need chronic blood transfusions […]
Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients
Therapeutic Candidate or Device HSC gene modified by a lentiviral vector which encodes a triple combination of HIV-resistance genes and a pre-selective marker. Indication HIV in AIDS-lymphoma patients. Therapeutic Mechanism Bone marrow transplants are standard of care for AIDS-lymphoma patients providing a cure for the patient's lymphoma. A purified population of triple combination anti-HIV lentiviral […]
A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of patients with severe X-linked Chronic Granulomatous Disease (XCGD) lacking matched donors. Therapeutic Mechanism Transplantation and engraftment of gene-corrected autologous HSPC after reduced intensity conditioning for […]
TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.
Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and chronic Graft versus Host Disease (GvHD) in patients undergoing mismatched stem cell transplant. Therapeutic Mechanism GvHD is driven by the reaction of allogenic donor T […]
Hematopoietic Stem Cell Gene Therapy for XCGD
Therapeutic Candidate or Device Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector Indication X-linked Chronic Granulomatous Disease Therapeutic Mechanism Lentiviral vector (LV) modification of autologous hematopoietic stem and progenitor cells (HSPCs) to restore physiologic gp91phox expression. We have developed a next-generation LV designed by bioinformatic-guided screening of […]