Cell Therapy News Volume 20.14 | Apr 22 2019

TOP STORY

Gene Therapy Restores Immunity in Infants with Rare Immunodeficiency Disease

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. [Press release from the National Institute of Allergy and Infectious Diseases discussing online prepublication in The New England Journal of Medicine] Press Release 1 | Abstract | Press Release 2

(Ranked by impact factor of the journal)

A Safe and Potent Anti-CD19 CAR T Cell Therapy

Scientists performed a Phase I trial of CD19-BBz(86) CAR T cell therapy in patients with B cell lymphoma. Complete remission occurred in 6 of 11 patients who each received a dose of 2×10⁸–4×10⁸ CD19-BBz(86) CAR T cells. [Nat Med] Abstract | Press Release

In Utero Gene Editing for Monogenic Lung Disease

Using a CRISPR fluorescent reporter system, investigators demonstrated that precisely timed in utero intra-amniotic delivery of CRISPR-Cas9 gene editing reagents during fetal development results in targeted and specific gene editing in fetal lungs. Pulmonary epithelial cells are predominantly targeted in this approach, with alveolar type 1, alveolar type 2, and airway secretory cells exhibiting high and persistent gene editing. [Sci Transl Med] Full Article | Press Release

Lentiviral Hemopoietic Stem/Progenitor Cell Gene Therapy for Treatment of Wiskott-Aldrich Syndrome: Interim Results of a Non-Randomized, Open-Label, Phase I/II Clinical Study

Researchers performed a non-randomized, open-label, Phase I/II clinical study in pediatric patients with severe Wiskott-Aldrich syndrome, defined by either WAS gene mutation or absent Wiskott-Aldrich syndrome protein expression or a Zhu clinical score of 3 or higher. [Lancet Haematol] Full Article

Targeting Pre-Existing Anti-Transgene T Cell Response for Effective Gene Therapy of Mucopolysaccharidosis I in the Mouse Model of the Disease

The authors treated mucopolysaccharidosis type-I mice with an artificial immunization protocol to mimic the enzyme replacement therapy effect in patients and they demonstrated that alpha-L-iduronidase (IDUA)-corrected hematopoietic stem cell engraftment was impaired in pre-immunized animals by IDUA-specific CD8⁺ T cells spared by pre-transplant irradiation. [Mol Ther] Abstract

Threshold for Pre-Existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation

Non-human-primates with pre-existing αAAV-Abs were used to investigate the impact of αAAV9-Abs levels on transduction efficiency of rAAV9 via systemic delivery. No significant differences were observed in vector genome biodistribution in animals with ≤1:400 total serum αAAV9-IgG compared to αAAV9-Ab-negative animals, following an IV rAAV9-hNAGLU^op injection. [Mol Ther Methods Clin Dev] Full Article

Anti-CD166/4-1BB Chimeric Antigen Receptor T Cell Therapy for the Treatment of Osteosarcoma

The ability of CD166.BBζ CAR-T cells to kill CD166⁺ osteosarcoma cells was evaluated in vitro and in vivo. Intravenous injection of CD166.BBζ CAR-T cells into mice resulted in the regression of the tumor with no obvious toxicity. [J Exp Clin Cancer Res] Full Article

Neural Stem Cell-Derived Small Extracellular Vesicles Attenuate Apoptosis and Neuroinflammation after Traumatic Spinal Cord Injury by Activating Autophagy

Investigators demonstrated that neural stem cell-small extracellular vesicles can significantly reduce the extent of spinal cord injury, improve functional recovery, and reduce neuronal apoptosis, microglia activation, and neuroinflammation in rats. [Cell Death Dis] Full Article

DNA-Encoded Bispecific T Cell Engagers and Antibodies Present Long-Term Antitumor Activity

Scientists describe the development of a designed synthetic DNA plasmid, which they optimized to permit high expression of an anti-HER2 antibody (HER2dMAb) and delivered it into animals through adaptive electroporation. HER2dMAb was efficiently expressed in vitro and in vivo, reaching levels of 50 μg/ml in mouse sera. [JCI Insight] Full Article

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Cell Therapy for Parkinson′s Disease Is Coming of Age: Current Challenges and Future Prospects with a Focus on Immunomodulation

The authors discuss the factors that affect the outcome of cell therapy in Parkinson’s disease, with focus on the cell types used and the functional effects of the host immune response on graft survival and differentiation. [Gene Ther] Abstract

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

Dystrogen Therapeutics’ Gene Therapy Shows Promise for Huntington’s

DYST 201, a gene therapy targeting the mutated forms of the gene responsible for Huntington’s disease, has shown promising results, Dystrogen Therapeutics announced. [Press release from Dystrogen Therapeutics (BioNews Services, LLC) discussing research to be presented at the 44^th Federation of European Biochemical Societies (FEBS) Congress and the 24^th Annual Meeting of the RNA Society, Krakow] Press Release

Celyad to Present Update on Allogeneic and Autologous NKG2D-Based CAR-T Candidates in Refractory mCRC

Celyad announced that clinical data from the SHRINK and alloSHRINK Phase I trials, evaluating the safety of NKG2D-based autologous and allogeneic CAR-T candidates, CYAD-01 and CYAD-101, respectively, will be presented. [Press release from Celyad discussing research to be presented at the European Society for Medical Oncology (ESMO) 21^st World Congress on Gastrointestinal Cancer (WCGIC), Barcelona] Press Release

Cell Medica Collaborator, Baylor College of Medicine, Showcasing Promise of CAR-NKT Therapies

Cell Medica announced that its collaborators from Baylor College of Medicine and Texas Children’s Hospital will be presenting the latest progress related to its innovative CAR therapy utilizing natural killer T cells (CAR-NKT). [Press release from Cell Medica discussing research to be presented at the 22^nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release

Axovant Announces Upcoming Presentations at the Annual Meeting of the American Society of Gene and Cell Therapy

Axovant Gene Therapies Ltd. announced two upcoming oral presentations and one poster presentation. [Press release from Axovant Gene Therapies Ltd. discussing research to be presented at the 22^nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), Washington, DC] Press Release

BioTime to Present Data from OpRegen^® Phase I/IIa Clinical Study

BioTime, Inc. announced that updated results from a Phase I/IIa clinical study of its lead product candidate, OpRegen^®, a retinal pigment epithelium cell transplant therapy currently in development for the treatment of dry age-related macular degeneration with geographic atrophy, will be presented. [Press release from BioTime, Inc. discussing research to be presented at the 2019 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Vancouver] Press Release

Vineti and Marker Therapeutics Announce Strategic Partnership to Support Diverse Cell Therapy Portfolio

Vineti Inc. announced a new partnership to advance and scale Marker Therapeutics’ T cell therapy pipeline. Marker Therapeutics is a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications. [Vineti Inc. (GlobeNewswire, Inc.)] Press Release

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced that the FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia. [CRISPR Therapeutics] Press Release

Precision BioSciences Announces Dosing of First Patient in Off-the-Shelf CAR T Cell Therapy Phase I/IIa Clinical Trial

Precision BioSciences announced it has dosed the first patient in the Phase I/IIa clinical trial of PBCAR0191, its first gene-edited allogeneic anti-CD19 CAR T cell product candidate. [Precision BioSciences] Press Release

Two Patients Treated with CRISPRed Cells in Immunotherapy Trial

Doctors have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania. A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. [The Scientist] Editorial

US Patent & Trademark Office Issues Patent on Anixa Biosciences CAR-T Cancer Treatment Technology

Anixa Biosciences, Inc. announced that the United States Patent and Trademark Office has issued US Patent No. 10,259,855. This is the first patent covering Anixa’s CAR-T cancer treatment technology. [Anixa Biosciences, Inc.] Press Release

FDA Agrees to Rolling Review of Mesoblast’s Biologic License Application for Its Cell Therapy in Children with Steroid Refractory Acute Graft versus Host Disease

Mesoblast Limited announced that the FDA has agreed that Mesoblast can submit on a rolling basis a Biologics License Application for its allogeneic cellular medicine remestemcel-L in children with steroid-refractory acute graft versus host disease. [Mesoblast Limited] Press Release

PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

Parent Project Muscular Dystrophy (PPMD) announced that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment for Duchenne. [Parent Project Muscular Dystrophy] Press Release

Sernova Announces First Transplantation of Therapeutic Cells in Cell Pouch for the Company Phase I/II U.S. Clinical Study for Diabetes

Sernova Corp. announced the first Cell Pouch™ transplant of insulin-producing cells in its US Phase I/II clinical trial evaluating the safety and efficacy of the cell pouch for clinical islet transplantation in type 1 diabetes. [Sernova Corp.] Press Release

Major U.S. Cancer Center Ousts ‘Asian’ Researchers after NIH Flags Their Foreign Ties

The MD Anderson Cancer Center has ousted three senior researchers after the U.S. National Institutes of Health (NIH) in Bethesda, Maryland, informed it that the scientists had committed potentially “serious” violations of agency rules involving confidentiality of peer review and the disclosure of foreign ties. [ScienceInsider] Editorial

How US-China Political Tensions Are Affecting Science

In the latest twist, several US universities are expected this month to announce the actions they have taken against foreign scientists caught breaking rules concerning National Institutes of Health funding, according to comments made by agency director Francis Collins to the Senate Appropriations Committee. [Nature News] Editorial

NEW European CanCer Organisation (ECCO) 2019: European Cancer Summit
September 12-14, 2019
Brussels, Belgium

Visit our events page to see a complete list of events in the community.

NEW Postdoctoral Associate – Cancer Immunology (Winship Cancer Institute)

Research Technologist – Immunology (STEMCELL Technologies Inc.)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

DPhil Scholarship – Retinal Gene Therapy (University of Oxford)

Gene Editing Specialist – Rare Genetic Disorders (Novo Nordisk)

Director of Research – Division of Translational Medicine & Human Genetics (University of Pennsylvania)

Postdoctoral Research Associate – Retinal Stem Cell Biology and Regeneration (University of Nebraska Medical Center)

Cell Biologist – Regenerative Medicine (Hepatx Corporation)

Associate Scientist – Immuno-Oncology (Hackensack Meridian Health Center for Discovery & Innovation)

Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center)

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

Faculty Positions – Hematology and Oncology (University of California, Davis)

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