Human Stem Cell Use: Adult or Tissue Stem Cell
Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa
Therapeutic Candidate or Device CNS10-NPC – a human neural progenitor cell line Indication Retinitis Pigmentosa Therapeutic Mechanism 1. Phagocytosis of photoreceptor outer segment debris. 2. The release of pro-survival factors that have localized diffusion to inhibit retinal photoreceptor cell death. 3. Immunomodulation resulting in markedly fewer host inflammatory cells at the site of CNS10-NPC engraftment […]
LADICell
Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Leukocyte Adhesion Deficiency-I (LAD-I). Indication The target clinical indication is Leukocyte Adhesion Deficiency-I (LAD-I), a pediatric hematological rare disease. Therapeutic Mechanism The therapeutic is based in an ex-vivo, lentiviral-based, patient-specific approach by genetically engineering the patient's own CD34 positively selected […]
A Phase I Study of Chimeric Antigen Receptor Engineered Stem/Memory T Cells for the Treatment of HER2-Positive Brain Metastases
Therapeutic Candidate or Device Autologous naïve-stem/memory T cells engineered with a chimeric antigen receptor targeting the HER2 antigen (HER2BBζ-Tn/mem) Indication HER2-positive brain and/or leptomeningeal metastases, primarily from breast cancer Therapeutic Mechanism The proposed therapy aims to provide a safe and effective treatment option for patients with HER2-positive cancers that have metastasized to the central nervous […]
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 […]
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized as a conditioning agent for selectively eliminating endogenous stem cells in pediatric SCID patients prior to CD34+CD90+ hematopoietic stem cell transplantation for repopulation of the […]
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized as a conditioning agent for selectively eliminating endogenous stem cells in pediatric SCID patients prior to CD34+CD90+ hematopoietic stem cell transplantation for repopulation of the […]
Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients
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 TLI and rATG, and given a haploidentical living donor hematopoietic progenitor cell transplant (HSCT) , along with in vitro expanded recipient Treg cells (what we […]
Genetic Modification of Stem Cells and T cells to Activate the Immune System to Target Solid Tumors
Therapeutic Candidate or Device Autologous Peripheral Blood Stem Cells expressing the NY-ESO-1 TCR and a suicide/reporter gene combined with T cells expressing the same TCR Indication Locally advanced (unresectable stage IIIc) or metastatic malignancies (stage IV) that are HLA A2.1 +, NY-ESO-1 +, solid tumors, including sarcomas Therapeutic Mechanism The administration of TCR transduced mature […]
AB-205-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-205
Therapeutic Candidate or Device AB-205 consists of genetically engineered CD31+ cells derived from Human Umbilical Vein tissue. Indication To ameliorate or accelerate recovery from toxicities related to high-dose chemo followed by HDT-ASCT for the treatment of lymphoma and other cancers. Therapeutic Mechanism E-CEL UVEC cells (the active ingredient in AB-205) work both via the secretion […]