Human Stem Cell Use: Adult or Tissue Stem Cell
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 […]
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome
Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) syndrome Therapeutic Mechanism Administration of autologous CD4LVFOXP3 that constitutively and stably express wild-type FOXP3 gene will replace the lack of function regulatory T […]
A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia
Therapeutic Candidate or Device Autologous Muscle Derived Progenitor Cells (AMDC) isolated from skeletal muscle biopsy Indication Subjects with dysphagia (swallowing difficulties) that develops following treatment for head and neck cancer Therapeutic Mechanism Autologous Muscle Derived Progenitor Cells differentiate to form new muscle fibers, engraft into existing myofibers, and have been shown to increase muscle diameter […]
A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas
Therapeutic Candidate or Device Neural stem cells that are genetically engineered to express a cancer-killing virus that specifically targets brain tumor cells Indication Recurrent brain tumors in adults: high grade gliomas (HGG), such as glioblastoma (GBM) Therapeutic Mechanism The neural stem cells will act as carriers to deliver a cancer-killing virus to brain tumors (at […]
PHASE 1, OPEN LABEL, DOSE-ESCALATION STUDY OF CRX100 IN PATIENTS WITH ADVANCED SOLID TUMORS
Therapeutic Candidate or Device Immune cells loaded with a cancer-killing virus that targets cancer tissue, not healthy tissue. Indication Advanced, refractory solid tumors: CRC, HCC, Osteosarcoma, NNN Breast, Ovarian, Gastric Therapeutic Mechanism The proposed therapy pairs activated immune cells with an adapted oncolytic virus that selectively infects and kills malignant cells. The result of this […]
Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor T Cells for Diffuse Intrinsic Pontine Gliomas and Spinal Diffuse Midline Glioma
Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting GD2 (GD2-CART) Indication Brain tumors in children and young adults: Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG) Therapeutic Mechanism Progenitor GD2-CART cells will recognize DIPG/DMG cancer cells expressing GD2, become activated, divide, and kill the […]
Phase 1/1b study of T-allo10 infusion after HLA-partially matched abdepleted-HSCT in children and young adults with hematologic malignancies.
Therapeutic Candidate or Device An immunotherapy cell product, T-allo10, that is enriched for specialized immune cells called type I regulatory T (Tr1) cells Indication Children and young adults with relapse/refractory acute leukemia receiving a specialized stem cell transplant, αβdepleted-HSCT Therapeutic Mechanism αβdepleted-HSCT has increased the number of patients who can safely receive transplants, however this […]
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 […]
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication Amyotrophic Lateral Sclerosis (ALS) Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor […]
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair
Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs […]