Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells and Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

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UC San Francisco researchers aim to re­pair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can…

Continue ReadingGene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells and Gene Therapy for Artemis-Deficient Severe Combined Immunodeficiency Using a Self-Inactivating Lentiviral Vector

Phase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus

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Children with type 1 diabetes (T1D) face lifelong struggles with controlling their blood sugar levels and, despite careful management, an increased risk of severe complications. Currently, there is no approved therapy…

Continue ReadingPhase 2 Safety and Efficacy Study of CLBS03 Autologous T-Regulatory Cells in Adolescents with Recent Onset Type 1 Diabetes Mellitus

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…

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

Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa

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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…

Continue ReadingPhase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa

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

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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…

Continue ReadingA Double-Blind, Controlled Ph 2b Study of the Safety and Efficacy of Modified Stem Cells in Patients with Chronic Motor Deficit from Ischemic Stroke

A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML

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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…

Continue ReadingA Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Optimal Dose-Finding Study of DCC-UCB in Adults Receiving High Dose Chemotherapy for AML