Cell Therapy News Volume 20.38 | Nov 18 2019

TOP STORY

Human Autologous iPSC-Derived Dopaminergic Progenitors Restore Motor Function in Parkinson’s Disease Models

Transplantation of dopaminergic progenitors into rodent models of Parkinson’s disease robustly restored motor dysfunction and reinnervated host brain, while showing no evidence of tumor formation or redistribution of the implanted cells. [J Clin Invest] Abstract | Graphical Abstract | Full Article

(Ranked by impact factor of the journal)

AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy

Scientists demonstrated X-linked myotubular myopathy transcriptome remodeling and dose-dependent normalization of gene expression after gene transfer and created metrics to pinpoint potential biomarkers of disease progression and correction. [Mol Ther] Abstract

Prophylactic In Vivo Hematopoietic Stem Cell Gene Therapy with an Immune Checkpoint Inhibitor Reverses Tumor Growth in Syngeneic Mouse Tumor Models

In in vivo hematopoietic stem/progenitor cell-transduced mice with implanted mouse mammary carcinoma tumors, after initial tumor growth, tumors regressed and did not recur. [Cancer Res] Abstract

Low-Dose Busulfan Reduces Human CD34⁺ Cell Doses Required for Engraftment in C-Kit Mutant Immunodeficient Mice

The authors assessed human CD45⁺ chimerism after transplanting varying doses of human CD34⁺ hematopoietic stem cells with or without busulfan (BU) pretransplant conditioning in c-kit mutant mice that did not require conditioning. They then tested a range of BU using 2×105 human CD34⁺ cells. [Mol Ther Methods Clin Dev] Abstract | Full Article

Dosing and Re-Administration of Intravenous Lentiviral Vector for In Vivo Gene Therapy: Studies in Rhesus Monkeys and ADA-Deficient Mice

In mice and monkeys with repeat administration of lentiviral vectors (LV), a strong anti-vector antibody response was shown in response to the second LV administration which resulted in LV inactivation. ADA-deficient mice administered three separate doses resulted in acute toxicity. [Mol Ther Methods Clin Dev] Abstract

Applicability, Safety and Biological Activity of Regulatory T Cell Therapy in Liver Transplantation

To investigate the safety, applicability and biological activity of autologous regulatory T cells adoptive transfer in humans, the authors conducted an open-label, dose escalation, Phase I clinical trial in liver transplantation. [Am J Transplant] Abstract

Adult Human Dermal Progenitor Cell Transplantation Modulates the Functional Outcome of Split-Thickness Skin Xenografts

Scientists examined whether cultured dermal progenitor cells (DPCs) could regenerate dermis within an split-thickness skin grafting (STSG) and improved overall graft function. Adult human DPCs were transplanted into a full-thickness skin wound in immune-compromised mice and closed with a human STSG. [Stem Cell Reports] Full Article | Graphical Abstract

Transplanted Interneurons Improve Memory Precision after Traumatic Brain Injury

GABAergic progenitors, derived from the embryonic medial ganglionic eminence, migrated long distances following transplantation into the hippocampus of adult mice with traumatic brain injury, functionally integrated as mature inhibitory interneurons and restore post-traumatic decreases in synaptic inhibition. [Stem Cell Reports] Full Article | Press Release

Hemophilia A Ameliorated in Mice by CRISPR-Based In Vivo Genome Editing of Human Factor VIII

Researchers describe a CRISPR/Cas9-based in vivo genome editing method, combined with non-homologous end joining, enabling permanent chromosomal integration of a modified human B domain deleted-F8 at the albumin locus in liver cells. [Sci Rep] Full Article

Physicochemically Tuned Myofibroblasts for Wound Healing Strategy

Investigators confirmed that physicochemically stimulated myofibroblasts promoted faster healing of the wound compared to the case with non-stimulated myofibroblasts through elevated matrix reconstruction in the mouse model. [Sci Rep] Full Article

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Clinical Translation of Pluripotent Stem Cell Therapies: Challenges and Considerations

Scientists describe completed or ongoing trials of pluripotent stem cells, discuss their potential mechanisms of action, and considers how to address the challenges required for them to become a major therapy, using heart repair as a case study. [Cell Stem Cell] Abstract

Combination Immunotherapy with CAR T Cells and Checkpoint Blockade for the Treatment of Solid Tumors

The authors review preclinical and clinical combination therapy with CAR T cells and checkpoint blockade agents, with a focus on solid tumor malignancies. [Cancer Cell] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Allogene Therapeutics and Notch Therapeutics Announce Collaboration to Research and Develop Induced Pluripotent Stem Cell (iPSC)-Derived Allogeneic Therapies for Hematologic Cancer Indications

Allogene Therapeutics, Inc. and Notch Therapeutics Inc. announced an exclusive worldwide collaboration and license agreement to research and develop iPSC AlloCAR^™ therapy products for initial application in non-Hodgkin lymphoma, leukemia and multiple myeloma. [Allogene Therapeutics, Inc.] Press Release

MD Anderson and Takeda Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform

The University of Texas MD Anderson Cancer Center and Takeda Pharmaceutical Company Limited announced an exclusive license agreement and research agreement to develop cord blood-derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies, ‘armored’ with IL-15, for the treatment of B-cell malignancies and other cancers. [The University of Texas MD Anderson Cancer Center] Press Release

bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous Lentiviral Vector Hematopoietic Stem Cell Gene Therapy

bluebird bio, Inc. and Forty Seven, Inc. announced that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen, FSI-174 plus magrolimab, with bluebird’s ex vivo lentiviral vector hematopoietic stem cell gene therapy platform. [bluebird bio, Inc.] Press Release

Editas Medicine and Celgene Corporation Amend Existing Collaboration to Develop and Commercialize Autologous and Allogeneic T Cell Medicines for the Treatment of Cancer and Autoimmune Diseases

Editas Medicine, Inc. announced an amended collaboration with Celgene Corporation under which the parties may research, develop, and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. [Editas Medicine, Inc.] Press Release

Sarepta and StrideBio Announce Multi-target Strategic Collaboration to Advance Novel Gene Therapies

Sarepta Therapeutics, Inc. and StrideBio, Inc. announced the signing of a collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system and neuromuscular targets. [Sarepta Therapeutics, Inc.] Press Release

Early Results from First-in-US Trial of CRISPR-Edited Immune Cells for Cancer Patients Suggest Safety of Approach

Researchers have infused three participants in the trial thus far – two with multiple myeloma and one with sarcoma – and have observed the edited T cells expand and bind to their tumor target with no serious side effects related to the investigational approach. [University of Pennsylvania] Press Release

Cartesian Initiates CAR-T Clinical Trial in Myasthenia Gravis

Cartesian Therapeutics announced that it has initiated a Phase I/II clinical trial of its lead CAR-T candidate, Descartes-08, in patients with generalized myasthenia gravis. [Cartesian Therapeutics] Press Release

Kiadis Pharma Changes Strategy to Focus Solely on Development of Natural Killer (NK) Cell Therapeutics and Terminates Development of ATIR101

Kiadis Pharma N.V. announced that it has completed a strategic portfolio review and has decided to change its strategy and focus all resources and investments on the company’s NK-cell therapy platform and product candidates. [Kiadis Pharma N.V.] Press Release

Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold

The FDA halts a study by Solid Biosciences after a patient experiences severe side effects following treatment. [The Scientist] Editorial

Ballot Initiative Takes Shape to Give California Stem Cell Agency a Second Life

As California’s stem cell research agency runs through the last of its $3 billion in state funding, a delicate negotiation is underway between its leadership and the man developing the 2020 ballot initiative to keep it alive. [ScienceInsider] Editorial

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