Cell Therapy News Volume 20.19 | Jun 3 2019

TOP STORY

Highly Efficient Editing of the β-Globin Gene in Patient-Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease

Gene-corrected sickle cell disease hematopoietic stem and progenitor cells retained the ability to engraft when transplanted into non-obese diabetic-SCID-gamma mice with detectable levels of gene correction 16–19 weeks post-transplantation. [Nucleic Acids Res] Full Article | Press Release

(Ranked by impact factor of the journal)

Gene-Edited Stem Cells Enable CD33-Directed Immune Therapy for Myeloid Malignancies

Scientists showed highly efficient genetic ablation of CD33 antigen using CRISPR/Cas9 technology in human stem/progenitor cells (HSPC) and provide evidence that the deletion of CD33 in HSPC doesn’t impair their ability to engraft and to repopulate a functional multilineage hematopoietic system in vivo. [Proc Natl Acad Sci USA] Full Article

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-Existing Anti-AAV5 NABs

Researchers showed that three of ten patients tested positive for anti-adeno-associated virus (AAV)5 neutralizing antibodies (NABs) using a more sensitive luciferase-based assay. However, no relationship was observed between the presence of pre-treatment anti-AAV5 NABs and the therapeutic efficacy of AMT-060. [Mol Ther Methods Clin Dev] Abstract | Full Article

Cell-Based Therapy Restores Olfactory Function in an Inducible Model of Hyposmia

The authors report the development of a novel mouse model of inducible hyposmia and demonstrated that purified tissue-specific stem cells delivered intranasally engraft to produce olfactory neurons, achieving recovery of function. [Stem Cell Reports] Full Article | Press Release

T Memory Stem Cells after Allogeneic Hematopoietic Cell Transplantation: Unique Long‐Term Kinetics and Influence of Chronic Graft‐versus‐Host Disease

Investigators conducted a cross‐sectional study to evaluate the proportions, absolute counts, phenotypes and functions of T memory stem cells in 152 adult patients without disease recurrence at least 12 months after undergoing hematopoietic cell transplantation. [Br J Haematol] Abstract

IGF-1 Overexpression Improves Mesenchymal Stem Cell Survival and Promotes Neurological Recovery after Spinal Cord Injury

The authors investigated whether bone marrow-derived mesenchymal stem cells (BMMSCs) overexpressing insulin-like growth factor 1 (IGF-1), a cytokine involved in neural development and injury repair, improved the therapeutic effects of BMMSCs in spinal cord injury. [Stem Cell Res Ther] Full Article

Telomerase Gene Therapy Ameliorates the Effects of Neurodegeneration Associated to Short Telomeres in Mice

Scientists describe a mouse model of neurodegeneration owing to the presence of short telomeres in the brain as the consequence of telomerase deficiency. They found similar signs of neurodegeneration in very old mice as the consequence of physiological mouse aging. [Aging (Albany NY)] Full Article

Long-Term Survival and Differentiation of Human Thymocytes in Human Thymus-Grafted Immunodeficient Mice

Immunodeficient mice were transplanted with human thymic tissue along with autologous GFP-expressing or allogeneic CD34⁺ cells and followed for human thymopoiesis and T-cell development from the thymic progenitors versus CD34⁺ cells, which can be distinguished by GFP or HLA expression. [Immunotherapy] Abstract

NES1/KLK10 and hNIS Gene Therapy Enhanced Iodine-131 Internal Radiation in PC3 Proliferation Inhibition

Results of in vitro and in vivo tumor proliferation experiments and ¹⁸F-fluorothymidine micro-positron emission tomography/computed tomography imaging showed that combined NES1 gene therapy and ¹³¹I radiation therapy mediated by overexpressed hNIS protein had the best tumor proliferative inhibition effect. [Front Med] Abstract

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Gene Editing for Immune Cell Therapies

Autologous T cells that have been genetically modified to express a chimeric antigen receptor targeting the B cell antigen CD19 have yielded remarkable clinical responses in patients with B cell malignancies, and are now on the market as anticancer ‘drugs’. [Nat Biotechnol] Abstract

The Next Generation of CRISPR-Cas Technologies and Applications

New advances are considerably improving the understanding of biological processes and are propelling CRISPR-Cas-based tools towards clinical use in gene and cell therapies. [Nat Rev Mol Cell Biol] Abstract

Principles of and Strategies for Germline Gene Therapy

The authors discuss novel strategies and provide a path forward for safe, high-efficiency germline gene therapy that may provide a promising new paradigm for preventing the passage of deleterious genes from parent to child. [Nat Med] Abstract

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City of Hope Doctors Present Research on First KRAS Inhibitor and CAR T Cell Therapy

A first-ever inhibitor of a cancer gene found in some lung, colorectal and other cancers and the effectiveness of chimeric antigen receptor (CAR) T cell therapy for patients with chronic lymphocytic leukemia are just some of the research topics that City of Hope physicians and scientists presented. [Press release from City of Hope discussing research presented at the American Society of Clinical Oncology (ASCO) 2019 Annual Meeting, Chicago] Press Release

Amicus Therapeutics and the University of Pennsylvania Announce Major Expansion of Gene Therapy Collaboration

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania announced a major expansion to their collaboration with rights to pursue collaborative research and development of novel gene therapies for lysosomal disorders and 12 additional rare diseases. [Amicus Therapeutics] Press Release

Rexgenero Successfully Expands Manufacturing of REX-001 through Partnership with German Red Cross Blood Donor Service

Rexgenero announced that it has successfully expanded manufacturing of its lead development candidate REX-001 through a partnership with the German Red Cross Blood Donor Service in Frankfurt, Germany, signed earlier this year. [Rexgenero] Press Release

Calidi Biotherapeutics Announces a Collaborative Study with the National Institutes Of Health (NIH) on the Therapeutic Potential of Oncolytic Viruses Delivered by Mesenchymal Stem Cells

Calidi Biotherapeutics, Inc. announced their ongoing scientific collaboration with David F. Stroncek, M.D., Chief, Cell Therapy Section at the Department of Transfusion Medicine, NIH to jointly study the impact of different stem cell carriers on the therapeutic potential of oncolytic viruses in the treatment of cancer. [Calidi Biotherapeutics, Inc. (PR Newswire Association LLC.)] Press Release

Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I

Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele announced that Orchard has been granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of Mucopolysaccharidosis Type I (MPS-I) developed by the San Raffaele-Telethon Institute for Gene Therapy. [Orchard Therapeutics] Press Release

BioMarin Announces that Phase III Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the US and Europe

BioMarin Pharmaceutical Inc. announced that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the US and Europe. [BioMarin Pharmaceutical Inc.] Press Release

Rejenevie™ Therapeutics Announces Start of Phase II Immune Restoration Treatment Clinical Trial

Rejenevie™ Therapeutics announced the commencement of the company’s Phase II clinical trial to study the safety and effectiveness of its proprietary AR-100 immune restoration therapy to boost the function of the aging immune system. [Rejenevie™ Therapeutics] Press Release

BrainStorm Announces Second Clinical Site to Enroll Patients in Its Progressive MS Phase II Study

BrainStorm Cell Therapeutics Inc. announced that the Stanford University School of Medicine will serve as the second contracted clinical study site for the company’s Phase II open-label, multicenter study of repeated intrathecal administration of autologous MSC-NTF cells in participants with progressive Multiple Sclerosis (MS). [BrainStorm Cell Therapeutics Inc.] Press Release

REGENXBIO Announces Completion of Dosing for Phase I/IIa Clinical Trial of RGX-314 in Wet AMD

REGENXBIO Inc. announced it completed dosing across all five cohorts in the Phase I/IIa clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). [REGENXBIO Inc.] Press Release

BioMarin Provides Three Years of Clinical Data from Ongoing Phase I/II Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase I/II study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. [BioMarin Pharmaceutical Inc.] Press Release

NantKwest Launches First-in-Human Targeted NK Phase I Clinical Trial of CD19 T-haNK Cell Therapy in Lymphoma Patients

NantKwest Inc. announced that the company’s t-haNK investigational new drug application has cleared FDA review and the program has now transitioned to a first-in-human clinical trial targeting CD19 t-haNK in advanced B-cell lymphoma. [NantKwest Inc.] Press Release

bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (Autologous CD34⁺ Cells Encoding βA-T87Q-globin Gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β⁰/β⁰ Genotype

bluebird bio, Inc. announced that the European Commission has granted conditional marketing authorization for ZYNTEGLO™, a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have a β⁰/β⁰ genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. [bluebird bio, Inc.] Press Release

Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala

Passage Bio announced that its third clinical trial program will be for infantile Krabbe disease, an inherited disease that causes progressive damage to the nervous system. [Passage Bio] Press Release

Ziopharm Oncology Announces Exclusive License with National Cancer Institute to Identify and Use T-Cell Receptors Targeting Neoantigens for Cancer with Sleeping Beauty Platform

Ziopharm Oncology, Inc. announced an exclusive licensing agreement with the National Cancer Institute for intellectual property for the development and commercialization of cell therapies for cancer. [Ziopharm Oncology, Inc.] Press Release

Fibrocell Receives FDA Regenerative Medicine Advanced Therapy Designation for FCX-007 Gene Therapy for the Treatment of RDEB

Fibrocell Science, Inc. announced that the FDA has granted the Regenerative Medicine Advanced Therapy designation to FCX-007, the company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB)—a devastating, genetic skin disease with high mortality. [Fibrocell Science, Inc.] Press Release

Mesoblast Initiates Rolling Submission of Biologics License Application (BLA) to US FDA for Remestemcel-L in the Treatment of Acute Graft versus Host Disease

Mesoblast Limited announced that it has filed the first component of a rolling submission for a BLA to the FDA for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease, a life-threatening complication of an allogeneic bone marrow transplant. [Mesoblast Limited] Press Release

Iovance Biotherapeutics Announces New Facility to Support US Production of Tumor Infiltrating Lymphocyte Cell Therapy Products

Iovance Biotherapeutics, Inc. announced that it has entered into a lease agreement to build an approximately 136,000 square foot commercial-scale production facility in Philadelphia for commercial and clinical production of autologous tumor-infiltrating lymphocyte products, including its candidate lifileucel. [Iovance Biotherapeutics, Inc.] Press Release

Gene Edits to ‘CRISPR Babies’ Might Have Shortened Their Life Expectancy

The scientist who edited the genomes of twin girls in an attempt to make them resistant to HIV might have inadvertently shortened their life expectancy. People with two disabled copies of the CCR5 gene — the version that protects against HIV infection — are 21% more likely to die before the age of 76 than are people with at least one working copy of the gene, according to a study published on 3 June in Nature Medicine. [Nature News] Editorial

US Science Academy Approves Plan to Oust Sexual Harassers

The US National Academy of Sciences has approved an amendment to its by-laws that allows its governing council to expel members for misconduct, including proven cases of sexual harassment. [Nature News] Editorial

NEW Keystone Symposia: Cancer Stem Cells: Advances in Biology & Clinical Translation
October 13-17, 2019
Hong Kong, China

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NEW Postdoctoral Research Fellowships – Kidney Disease (Vanderbilt University Medical Center)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University)

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Postdoctoral Research Fellow – Translational Gene Therapy (Fred Hutchinson Cancer Research Center)

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

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)

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

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