Therapeutic/Technology: Therapeutic Approach
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]