Therapeutic/Technology: Gene-modified, personalized cell therapy
Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors
Therapeutic Candidate or Device Autologous chimeric antigen receptor T cells derived from naive/memory T cells and engineered to target IL13Rα2 on pediatric malignant brain tumors. Indication Recurrent/refractory malignant pediatric brain tumors that express the tumor-associated antigen IL13Rα2. Therapeutic Mechanism Naive and memory T cells are harvested from patients and reprogrammed to express chimeric antigen receptors […]
A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Infantile Malignant Osteopetrosis (IMO). Indication The target clinical indication is Infantile Malignant Osteopetrosis (IMO), a pediatric, autosomal recessive rare disease. Therapeutic Mechanism The therapeutic mechanism is based on an ex-vivo, lentiviral-based, patient-specific approach by genetically engineering the patient's own CD34+ […]
Anti-HIV duoCAR-T cell therapy for HIV infection
Therapeutic Candidate or Device HIV-specific CAR-T cells Indication Management of HIV infection Therapeutic Mechanism We will modify T cells such that they are able to directly control HIV in the absence of therapy. Should this work, these cells will result in long-term control of HIV in absence of any ongoing treatment, a version of a […]
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 […]
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 […]