Therapeutic/Technology: Therapeutic Approach


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 […]

A Phase 1 Study of FT819 in Participants with Moderate to Severe Active Systemic Lupus Erythematosus

Therapeutic Candidate or Device iPSC-derived CD19 CAR T cell therapy Indication Systemic Lupus Erythematosus Therapeutic Mechanism Depletion of CD19+ B cells and plasmablasts Unmet Medical Need Potential for drug-free remission and reset of the immune system Project Objective Phase 1 trial completed Major Proposed Activities Site Activation and Patient Enrollment Clinical Trial Data Monitoring, Database […]

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 […]

The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

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 […]

A Phase 2b Study of the Efficacy of a Novel Pro-Neurogenesis/Pro-Plasticity Drug for Bipolar Depression Using a Precision Psychiatry Approach

Therapeutic Candidate or Device ALTO-100 Indication Bipolar depression (I or II) Therapeutic Mechanism ALTO-100 increased hippocampal neurogenesis and neuroplasticity. Its effects are mediated by signaling through the brain derived neurotrophic factor (BDNF) pathway. Unmet Medical Need Bipolar depression is a severe, life-long disorder with high burden of illness and risk of suicide. The only approved […]

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 […]

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 […]

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 […]

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 […]