Program Type: Clinical
A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19
Therapeutic Candidate or Device Human placental hematopoietic stem cell derived natural killer cells (CYNK-001) Indication SARS-CoV-2 positive patients requiring hospital admission and have any 2 out of 3 symptoms: fever, cough, or positive disease-related chest x-ray. Therapeutic Mechanism CYNK-001 is allogenic, human placental hematopoietic stem cell-derived NK cells that express the dominant NK cells marker […]
Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)
Therapeutic Candidate or Device Novel testing of a cell based therapy (Mesenchymal Stromal Cells) for respiratory failure from ARDS. Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will hopefully improve oxygenation in ARDS patients and potentially decrease mortality. Unmet Medical Need There is an unmet need for more effective treatments for ARDS both […]
Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use
Therapeutic Candidate or Device The therapeutic candidate is COVID-19 convalescent plasma (CCP) Indication The target indication is treatment of severe COVID-19 infection Therapeutic Mechanism Neutralizing antibodies are part of the humoral response of the adaptive immune system against viruses, and can be detected in plasma of convalescent individuals. Transfusion of COVID-19 convalescent plasma can provide […]
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle globin and induction of anti-sickling fetal hemoglobin Unmet Medical Need Sickle cell is a severe disease with protean manifestations. The only curative therapy is an […]
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial Therapeutic Mechanism Blood stem cells are harvested […]
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Therapeutic Candidate or Device ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients. Indication Severe Sickle Cell Disease Therapeutic Mechanism Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the […]
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 […]
Selective, Off-the-Shelf Logic Gated CAR NK Cell Therapy Targeting CD33 and/or FLT3 Expressing Hematologic Malignancies
Therapeutic Candidate or Device SENTI-202 is an allogeneic off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cellular therapy targeting CD33 and/or FLT3 malignancies. Indication CD33 and/or FLT3 expressing hematologic malignancies, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Therapeutic Mechanism SENTI-202 has been designed to incorporate a logic gated gene circuit and an engineered […]
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 […]