Therapeutic/Technology: Gene-modified, personalized cell therapy
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Stem Cell-Based iNKT Cell Therapy for Cancer
Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are lacking existing effective treatments. Mechanism of Action The proposed candidate will generate therapeutic levels of invariant natural killer T (iNKT) cells in cancer patients, helping […]
Process development for establishing an iPSC-based therapeutic candidate for Canavan disease
Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This candidate has the potential to develop into a therapy for Canavan disease, a fatal neurological disease that has no cure or standard treatment. Mechanism of Action The proposed candidate is […]
Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease
Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs and Sandhoff disease patients who have no curative or ameliorating treatment. Mechanism of Action Wild type HexA and HexB will be delivered to affected neurons […]
A phase 1/2 study to evaluate a bispecific CD19/CD20-directed CAR T cell, in refractory lupus nephritis and systemic lupus erythematosus
Therapeutic Candidate or Device IMPT-514 is an autologous T-cell product genetically modified to express a chimeric antigen receptor targeting CD19 and CD20 Indication Active, refractory lupus nephritis (LN) and systemic lupus erythematosus (SLE) Therapeutic Mechanism Targeted depletion of pathogenic autoantibody-secreting B cells as well as CD20+ T cells by a bispecific CD19/CD20 -directed CAR T […]
Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis
Therapeutic Candidate or Device NXC-201 Indication AL Amyloidosis Therapeutic Mechanism Genetically modified T-cell targeting B-cell maturation antigen (BCMA) Unmet Medical Need Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma cell disorder that results in organ deposition. In the US, there are ~3,972 diagnoses every year. Currently, there is no cure for AL amyloidosis. Project […]
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 […]
Phase I Trial of Locoregionally Delivered Autologous B7-H3 CAR T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme
Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting B7-H3 (B7-H3CART) Indication Brain tumors in adults: Glioblastoma Multiforme (GBM) Therapeutic Mechanism Progenitor B7-H3CART cells will recognize GBM cancer cells expressing B7-H3, become activated, divide, and kill the cancer cells Unmet Medical Need Glioblastoma Multiforme (GBM) is the most […]
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 […]