Therapeutic/Technology: Gene-modified, personalized cell therapy
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 […]
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 […]
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children with severe combined immunodeficiency or "bubble baby disease" due to a defective gene that makes a protein called Artemis Therapeutic Mechanism Stem cells from the […]
A phase I trial of intratumoral administration of CCL21-gene modified dendritic cell (DC) combined with intravenous pembrolizumab for advanced NSCLC
Therapeutic Candidate or Device Combination therapy with adenoviral CCL21 gene-modified DC and pembrolizumab Indication Patients with confirmed and measurable stage IV NSCLC expressing PD-L1 in less than 50% of cells who are naïve to systemic treatment for NSCLC. Therapeutic Mechanism The central rationale this approach is to utilize in situ vaccination with intratumoral injection of […]
Clinical Study of T stem cell memory (Tscm)-based CAR-T cells in Patients with Multiple Myeloma
Therapeutic Candidate or Device Genetically engineered, Centyrin-based, stem cell memory CAR-T cells (CARTyrin T cells) Indication Multiple Myeloma Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that are removed from a myeloma patient's body and genetically engineered to express a receptor that binds to BCMA that is selectively found on myeloma cells, […]
Phase I Study of Chimeric Antigen Receptor Engineered Central Memory T cells for the Treatment of Malignant Glioma
Therapeutic Candidate or Device A promising immunotherapy utilizing a patient’s memory T cells engineered to express chimeric antigen receptors for targeted tumor killing. Indication Malignant glioma (WHO III and IV), including glioblastoma (WHO IV), that express the tumor-associated antigen IL-13 receptor alpha 2 (IL13Rα2). Therapeutic Mechanism A promising immunotherapy utilizing a patient’s memory T cells […]
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 […]
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects
Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase – Severe Combined Immunodeficiency (or ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects
Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase – Severe Combined Immunodeficiency (or ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]