Disease Focus: Blood Cancer

BCMA/CS1 Bispecific CAR-T Cell Therapy to Prevent Antigen Escape in Multiple Myeloma

Translational Candidate A single-chain bispecific chimeric antigen receptor (CAR) targeting BCMA and CS1 will be used to in autologous T-cell therapy for multiple myeloma. Area of Impact Translational candidate will enable treatment of patients with heterogeneous or BCMA– multiple myeloma and prevent cancer relapse due to antigen loss. Mechanism of Action BCMA and CS1 are […]

Developing engineered autologous leukemia vaccines to target residual leukemic stem cells

Translational Candidate A universally applicable, patient-specific leukemia vaccine engineered to express a novel immune stimulatory combination for post-remission therapy Area of Impact There is a critical and unmet need for new and safe treatment for older acute myelogenous leukemia (AML) patients whose current prognosis is poor Mechanism of Action In older patients with AML, treatment […]

Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment

Translational Candidate Human embryonic stem cell (hESC)-derived natural killer (NK) cells to target relapsed/refractory Acute Myelogenous Leukemia (AML) Area of Impact hESC-derived NK cells provide a novel and potent approach to treat relapsed or refractory AML that is resistant to current chemotherapy options. Mechanism of Action hESC-derived NK cells provide a standardized, homogeneous, off-the-shelf cellular […]

A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia

Translational Candidate 17S-FD-895 is a potent small molecule splicing modulator that inhibits aberrant splicing in CSCs that have deregulated SF3B1 expression. Area of Impact Development of 17S-FD-895 could address a major bottleneck to reducing AML mortality by targeting splicing deregulated-CSCs that fuel AML relapse. Mechanism of Action 17S-FD-895 will positively impact patients with AML by […]

Development of ROR1 CAR-T cells to target cancer stem cells in advanced malignancies

Translational Candidate Autologous ROR1 CAR-T cell transduced with a lentiviral vector containing scFv (cirmtuzumab) with CD28, CD137, CD3zeta signaling domains Area of Impact ROR1 expressing cancer stem cells in solid tumors and hematologic tumors Mechanism of Action ROR1 CAR is a 3rd generation chimeric construct with an internal endodomain that transmits a CD3 zeta signal […]

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 […]

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 […]

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 […]

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 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML

Therapeutic Candidate or Device IO-202, a first-in-class antibody targeting leukocyte immunoglobulin-like receptor B4 (LILRB4), an immune inhibitory receptor Indication Acute myeloid leukemia (AML) with monocytic differentiation and chronic myelomonocytic leukemia (CMML) Therapeutic Mechanism IO-202 is the first T-cell activator for AML. Preclinical studies showed that IO-202 can convert a “don’t kill me” to “kill me” […]