Disease Focus: Leukemia, Acute Myeloid (AML)
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 […]
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 […]
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” […]
Phase 1b Trial of Hu5F9-G4 Monotherapy or Hu5F9-G4 in Combination with Azacitidine in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome
Therapeutic Candidate or Device Hu5F9-G4 is a drug called an antibody that is designed to mobilize the body's immune system to eliminate cancer and cancer stem cells. Indication Patients with relapsed and/or refractory AML or newly diagnosed AML who cannot receive standard high dose chemotherapy. Therapeutic Mechanism This treatment targets cancer stem cells, which are […]
A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML
Therapeutic Candidate or Device Cryopreserved, universal donor hematopoietic stem cell therapy that restores blood cell function and protects against infection after chemotherapy Indication Neutropenia arising from high-dose chemotherapy for treatment of Acute Myeloid Leukemia Therapeutic Mechanism The primary treatment for patients with AML is chemotherapy. Most chemotherapy results in a period of neutropenia (very low […]
Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor
Therapeutic Candidate or Device Rebecsinib is a novel small molecule inhibitor of ADAR1 splicing that selectively eradicates therapy-resistant cancer stem cells in blood cancers. Indication The target indication is relapsed/refractory secondary acute myeloid leukemia (sAML), or int-2 or high-risk myelofibrosis (MF). Therapeutic Mechanism Rebecsinib therapy for patients with relapsed/refractory sAML, or int-2 or HR MF […]
Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells
Therapeutic Candidate or Device TriLeukeVax, an autologous AML vaccine designed to stimulate induction of anti-leukemic cytolytic activity and improve relapse free survival (RFS). Indication Older leukemia patients who achieve remission with chemotherapy and are at high risk of relapse, but are not eligible for allogeneic transplantation Therapeutic Mechanism Most older patients with acute myelogenous leukemia […]