Cell Line Generation: Adult or Tissue Stem Cell
Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension
Translational Candidate Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells Area of Impact Pulmonary Arterial Hypertension (PAH), initially associated with Scleroderma (Systemic Sclerosis -SSC), and then applied to other causes of PAH Mechanism of Action Myeloperoxidase (MPO) protein produced by neutrophils plays a critical role in the development of PAH. Disrupting the MPO gene in […]
Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant
Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological diseases Therapeutic Mechanism 1. By using haploidentical parents, we will expand the number of potential living donors, and 2. with pre-HSCT immune ablation we can […]
Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Therapeutic Candidate or Device Autologous cultivated limbal stem cells (cLSC) Indication Limbal stem cell deficiency Therapeutic Mechanism Restoration of a normal corneal surface using cLSC might be achieved by replenishing the LSC population and/or providing trophic factors to stimulate residual LSCs. Unmet Medical Need Therapy using cultivated LSC, which achieves the best clinical outcomes is […]
A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Therapeutic Candidate or Device Allogeneic human retinal progenitor cells (hRPC) Indication Retinitis Pigmentosa (RP) Therapeutic Mechanism The cells are intended to remain suspended in the vitreous cavity of the eye and exert a beneficial neurotrophic effect on the degenerating retina. Unmet Medical Need RP is an incurable orphan disease. There are no treatments currently available […]
A Phase 1/2 Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous HSPC Transplant in Transfusion-dependent β-Thalassemia
Therapeutic Candidate or Device ST-400 is a gene-edited cell therapy candidate for patients with transfusion-dependent beta-thalassemia Indication Transfusion-dependent beta-thalassemia Therapeutic Mechanism ST-400 is intended to disrupt BCL11A erythroid enhancer in CD34+ HSPC resulting in an increase in fetal hemoglobin which can substitute for reduced or absent adult Hb. Therefore, treatment with ST-400 may potentially reduce […]
Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies
Therapeutic Candidate or Device T cells genetically engineered to express as bispecific Chimeric Antigen Receptor (CAR) targeting CD19 and/or CD22 Indication Patients with relapsed and refractory B cell malignancies Therapeutic Mechanism T cells expressing the bispecific CAR will recognize cancer cells expressing one of both of the target antigens. Upon recognition, the T cells will […]
Cellular Immunotherapy for Induction of Immune Tolerance in HLA Matched Living Donor Kidney Transplant Recipients
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 in HLA matched kidney transplants recipients Therapeutic Mechanism Following infusion and engraftment of MDR-101, the progeny cells establish a state of mixed lympo-hematopoetic chimerism. This […]
AB-110-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-110
Therapeutic Candidate or Device AB-110 consists of cord blood derived hematopoietic stem and progenitor cells co-cultured and expanded with E-CEL UVEC cells Indication Hematologic and immune reconstitution in patients who have received myeloablation conditioning Therapeutic Mechanism Stem and progenitor cells (active ingredient) of AB-110 engraft into the bone marrow of patients, rebuilding a new blood […]
Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Therapeutic Candidate or Device Allogeneic human retinal progenitor cells (hRPC) Indication Retinitis Pigmentosa (RP) Therapeutic Mechanism The cells are intended to remain suspended in the vitreous cavity of the eye and exert a beneficial neurotrophic effect on the degenerating retina. Unmet Medical Need RP is an incurable orphan disease. There are no treatments currently available […]
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 […]