Stage of Program: Clinical Trial, Phase 2
Allogeneic Cardiac-Derived Stem Cells for Patients Following a Myocardial Infarction
In this trial, Capricor Therapeutics used donor cells derived from the heart to treat patients at risk for developing heart failure after a heart attack. In previous clinical studies, the cells appeared to reduce scar tissue, promote blood vessel growth and improve heart function. This trial is ending after failing to achieve its primary goal […]
Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects
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 UCLA is using a patient’s own blood stem cells to try and rebuild the damaged immune systems of patients with ADA-SCID. They will use what’s […]
Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to repair the damage caused by this vision destroying disease. The cells are injected into the back of the eye and it’s hoped they will help […]
A Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke
Stroke is a major cause of long-term disability and there are no proven medical treatments for chronic stroke. SanBio is carrying out a Phase 2 clinical trial using mesenchymal stem cells (MSCs) to help people suffering from chronic disability following a stroke. The MSCs are isolated from the bone marrow of healthy adult donors, and […]
A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML
Nohla Therapeutics is testing a hematopoietic stem cell and progenitor cell therapy called NLA101 to help patients suffering from neutropenia, a condition that leaves people susceptible to deadly infections, after receiving chemotherapy for acute myeloid leukemia (AML). The company is currently launching a Phase 2 trial to test this treatment in adult AML patients that […]
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 […]
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 […]
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 […]
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 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy consisting of a recombinant AAV9 vector engineered with genetic elements designed to ensure consistent delivery and expression of the LAMP2B transgene. The therapy is administered […]