Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use

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Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections.  Blood plasma from patients that have recovered from COVID-19,…

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Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

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Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood.  Patients inherit defective copies of a gene called CTNS,…

Continue ReadingPhase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

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Researchers at the California Institute for Biomedical Research (CALIBR) have been awarded $8.447 million to test KA34, a drug that, in preclinical tests, recruits stem cells to create new cartilage…

Continue ReadingEvaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

Clinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors

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A team at Stanford University is using a molecule known as an antibody to target cancer stem cells. This antibody can recognize and bind to CD47, a protein the cancer stem cells…

Continue ReadingClinical Investigation of a Humanized Anti-CD47 Antibody in Targeting Cancer Stem Cells in Hematologic Malignancies and Solid Tumors

A Phase I dose escalation and expansion clinical trial of the novel first-in-class Polo-like Kinase 4 (PLK4) inhibitor, CFI-400945 in patients with advanced solid tumors

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Solid tumors are the most prevalent form of cancer, and are a major cause of death worldwide.  A team at UCLA is testing a drug for the treatment of cancer…

Continue ReadingA Phase I dose escalation and expansion clinical trial of the novel first-in-class Polo-like Kinase 4 (PLK4) inhibitor, CFI-400945 in patients with advanced solid tumors

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

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In ADA-SCID, allogeneic hematopoietic (blood) stem cell transplants from non-matched sibling donors are a high risk procedure. Additionally, the efficacy of chronic enzyme replacement therapy is uncertain in the long-term. A team at…

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Lentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning

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St. Jude Children's Research Hospital is teaming up with UC San Francisco to repair the damaged immune system of children born with SCID. They will genetically modify the patient's own…

Continue ReadingLentiviral Gene Therapy for Infants with X-linked Severe Combined Immunodeficiency using Autologous Bone Marrow Stem Cells and Busulfan Conditioning