Project Objective: Clinical Trial, Phase 1 or Phase 1/2
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 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
Therapeutic Candidate or Device Autologous stem cell-derived dopaminergic neuron replacement therapy. Indication Parkinson’s disease Therapeutic Mechanism The investigational cell therapy product is being studied as an autologous neuron replacement for Parkinson’s disease. Unmet Medical Need PD is a neurodegenerative disorder, affecting 1+ million people in the US. PD causes loss of dopamine (DA) neurons in […]
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 […]
A Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma
Therapeutic Candidate or Device DB107 (Toca 511), a retroviral replicating vector expressing yeast cytosine deaminase, which converts an antifungal prodrug to an anticancer drug Indication Newly diagnosed high-grade glioma Therapeutic Mechanism Retroviral replicating vector DB107 (Toca 511) spreads through tumors and delivers yeast cytosine deaminase (CD), which converts the antifungal prodrug 5-fluorocytosine (5-FC) to the […]
RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration
Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves loss of the RPE cells that support the overlying retina and vision. We transplant laboratory grown RPESC-RPE-4W cell to replace the RPE cells lost in […]
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 […]
Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia
Therapeutic Candidate or Device Immune T cells from a patient’s transplant donor engineered to express chimeric antigen receptors for targeted leukemia killing Indication Relapsed or refractory acute myeloid leukemia Therapeutic Mechanism Upon adoptive transfer, patient specific immune T cells that express chimeric antigen receptors will specifically recognize and directly kill leukemia cells expressing CD33. Unmet […]
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation in the SCN2A gene with both GOF and LOF effects when expressed in cells. nL-SCN2A-002 is designed to specifically bind the pathogenic allele and lower […]
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 […]
Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV
Therapeutic Candidate or Device Cytomegalovirus (CMV)-specific T cells that express a chimeric antigen receptor (CAR) which targets and eliminates HIV-infected cells Indication Management of human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome (AIDS) Therapeutic Mechanism Persons with HIV (PWH) lose immunity because of decreased T lymphocyte function. We propose to treat them with CAR T cells which […]