Human Stem Cell Use: Somatic Cell
CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia
Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options Mechanism of Action The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed […]
MPS II: Plasma cell delivery of iduronate sulfatase
Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II, a rare genetic disease causing multi-organ symptoms and death by age 15, if not treated. Current treatment does not address major symptoms. Mechanism of Action […]
Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy
Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a fatal muscle wasting disease with no cure. Mechanism of Action Our therapy uses CRISPR/Cas9 gene editing to permanently remove a hotspot region of DMD patient […]
Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma
Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]
Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma
Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]
Phase 1 Study of Autologous E-SYNC T Cells in Adult Participants with EGFRvIII+ Glioblastoma
Therapeutic Candidate or Device Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Indication Patients with MGMT unmethylated newly diagnosed GBM (Cohort 1), and patients for whom re-resection of recurrent GBM (Cohort 2). Therapeutic Mechanism In our proposed system, the first antigen EGFRvIII, which is expressed exclusively but heterogeneously […]
A PHASE 1B STUDY EVALUATING THE SAFETY AND EFFICACY OF AN ALLOGENEIC CELL THERAPY IN SUBJECTS WITH CLEAR CELL RENAL CELL CARCINOMA (ccRCC)
Therapeutic Candidate or Device An allogeneic anti-CD70 CAR-T cell product will be evaluated for the treatment of advanced or metastatic clear cell renal cell carcinoma (ccRCC). Indication Advanced or metastatic clear cell renal cell carcinoma (ccRCC) Therapeutic Mechanism The product is an allogeneic TRAC/CD52-knockout CAR T cell therapy targeting CD70. In this clinical study, the […]
Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors
Therapeutic Candidate or Device Adult Stem-Like T cells engineered with chimeric antigen receptor (CAR) to target cancers expressing IL13Ra2, including melanoma. Indication Advanced cancers that express IL13Ra2, including melanoma. Therapeutic Mechanism When administered to a patient, the engineered T cells will circulate through the blood and tissues to nd, recognize and kill tumor cells that […]
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1
Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication Mucopolysaccharidosis I (MPSI) is a rare disease that affects predominantly children. Untreated, these patients typically die by the age of 10. Therapeutic Mechanism The therapeutic […]
AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205
Therapeutic Candidate or Device AB-205 is an intravenously infused medicine containing genetically modified human vascular (endothelial) cells intended to repair damaged tissue Indication Treatment to reduce/prevent severe multi-organ complications from curative high-intensity cancer treatments involving stem cell transplantation Therapeutic Mechanism AB-205 acts through paracrine effects from expression of a rich mixture of reparative proteins called […]