Cell Therapy News Volume 18.41 | Dec 11 2017

TOP STORY

One-Dose Gene Therapy Produces Clotting Factor, Safely Stops Bleeding in Hemophilia B Patients

Researchers report positive results in a Phase I/II clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment enabled adult participants to safely produce sustained levels of clotting factor that prevented debilitating bleeding episodes. [Press release from The Children’s Hospital of Philadelphia discussing online prepublication in The New England Journal of Medicine] Press Release | Abstract | Editorial 1 | Editorial 2

(Ranked by impact factor of the journal)

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

In this multicenter study, patients with refractory large B-cell lymphoma who received CAR T-cell therapy with axicabtagene ciloleucel had high levels of durable response, with a safety profile that included myelosuppression, the cytokine release syndrome, and neurologic events. [N Engl J Med] Full Article | Press Release 1 | Press Release 2

Chimeric Antigen Receptor T Cells in Refractory B-Cell Lymphomas

The authors used autologous T cells that express a CD19-directed CAR to treat patients with diffuse large B-cell lymphoma or follicular lymphoma that had relapsed or was refractory to previous treatments. High rates of durable remission were observed, with recovery of B cells and immunoglobulins in some patients. [N Engl J Med] Full Article | Press Release 1 | Press Release 2

Runx3 Programs CD8⁺ T Cell Residency in Non-Lymphoid Tissues and Tumors

Scientists showed that mouse tissue-resident memory CD8⁺ T (T_RM) precursor cells represent a unique CD8⁺ T cell subset that is distinct from the precursors of circulating memory cell populations at the levels of gene expression and chromatin accessibility. Using computational and pooled in vivo RNA interference screens, they identified the transcription factor Runx3 as a key regulator of T_RM cell differentiation and homeostasis. [Nature] Abstract | Press Release

In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-Epigenetic Modulation

Researchers report a robust system for in vivo activation of endogenous target genes through trans-epigenetic remodeling. Results demonstrated that CRISPR/Cas9-mediated target gene activation can be achieved in vivo, leading to measurable phenotypes and amelioration of disease symptoms. [Cell] Abstract | Press Release | Editorial | Graphical Abstract

In Vivo Transplantation of 3D Encapsulated Ovarian Constructs in Rats Corrects Abnormalities of Ovarian Failure

The authors describe the in vivo performance of 3D bioengineered ovarian constructs that recapitulate native cell–cell interactions between ovarian granulosa and theca cells as an approach to cell-based hormone replacement therapy. [Nat Commun] Full Article | Press Release

Human Neural Stem Cell Transplantation Rescues Functional Deficits in R6/2 and Q140 Huntington’s Disease Mice

Researchers evaluated efficacy of transplanting a good manufacturing practice-grade human embryonic stem cell-derived neural stem cell line into striatum of Huntington’s disease (HD) modeled mice. In HD fragment model R6/2 mice, transplants improved motor deficits, rescued synaptic alterations, and were contacted by nerve terminals from mouse cells. [Stem Cell Reports] Full Article

IL-11 Promotes the Treatment Efficacy of Hematopoietic Stem Cell Transplant Therapy in Aplastic Anemia Model Mice through a NF-κB/MicroRNA-204/Thrombopoietin Regulatory Axis

The authors found that decreased miR-204-5p levels were coincident with increased proliferation in mouse hematopoietic stem cells (HSCs) following exposure to interleukin-11 (IL-11) in vitro. Consistent with these in vitro findings, IL-11 promoted HSC engraftment in a mouse model of aplastic anemia, an effect that was attenuated in cells overexpressing miR-204-5p. [Exp Mol Med] Full Article

Multiple Autologous Bone Marrow-Derived CD271⁺ Mesenchymal Stem Cell Transplantation Overcomes Drug-Resistant Epilepsy in Children

Investigators assessed the safety, feasibility, and potential efficacy of autologous bone marrow cell transplantation in pediatric patients with drug-resistant epilepsy. The therapy resulted in neurological and cognitive improvement in all patients, including a reduction in the number of epileptic seizures and an absence of status epilepticus episodes. [Stem Cells Transl Med] Full Article

Bone Marrow Transplantation Improves Motor Activity in a Mouse Model of Ataxia

Scientists used a model of ataxia, the PCD mutant mouse, to study the effect of healthy bone marrow transplantation on the restoration of defective mobility. Their results demonstrated that the transplant of wild-type bone marrow restores the mobility of PCD mice, increasing their capabilities of movement, exploration, speed, and motor coordination. [J Tissue Eng Regen Med] Abstract

Intracerebroventricular Delivery of Hematopoietic Progenitors Results in Rapid and Robust Engraftment of Microglia-Like Cells

Researchers demonstrated that transplantation of hematopoietic stem and progenitor cells in the cerebral lateral ventricles provides rapid engraftment of morphologically, antigenically, and transcriptionally dependable microglia-like cells. [Sci Adv] Full Article | Press Release

Cell Therapy for Critical Limb Ischemia: An Integrated Review of Pre-Clinical and Clinical Studies

Understanding how stem cells co-ordinate the myriad of cells and signals required for stable revascularization remains the key to translating the potential of stem cells into curative therapies for critical limb ischemia (CLI). Thus, combination delivery of multiple cell types within supportive bioengineered matricies may represent a new direction to improve cell therapy strategies for CLI. [Stem Cells] Abstract

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

St. Jude Gene Therapy Improves Immunity in Babies with ‘Bubble Boy’ Disease

Early evidence suggests that gene therapy developed at St. Jude Children’s Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined immunodeficiency disorder. [Press release from St. Jude Children’s Research Hospital discussing research presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

Celgene Corporation and bluebird bio Announce Updated Results from Ongoing Multicenter Phase I Study of bb2121 Anti-BCMA CAR T Cell Therapy in Patients with Late Stage Relapsed/Refractory Multiple Myeloma

Celgene Corporation and bluebird bio, Inc. announced that updated results from the ongoing CRB-401 Phase I clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 21 patients with late-stage relapsed/refractory multiple myeloma were presented in an oral presentation. [Press release from bluebird bio, Inc. discussing research presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

bluebird bio Presents New Data from Clinical Studies of LentiGlobin^™ Gene Therapy in Transfusion-Dependent β-Thalassemia

bluebird bio, Inc. announced data from two studies of its LentiGlobin gene therapy product candidate in patients with transfusion-dependent β-thalassemia. [Press release from bluebird bio, Inc. discussing research presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

CRISPR Therapeutics Announces Oral Presentation of New Data on CTX001, a CRISPR Gene-Edited Medicine for β-Thalassemia and Sickle Cell Disease

CRISPR Therapeutics announced that Dr. Bill Lundberg, Chief Scientific Officer of CRISPR, presented new data on CTX001, an investigational CRISPR gene-edited therapy for patients suffering from β-thalassemia and sickle cell disease. [Press release from CRISPR Therapeutics discussing research presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

ZIOPHARM Announces Presentation of Data from T-Cell Therapy Programs

ZIOPHARM Oncology, Inc. announced data supporting its non-viral approach to rapid manufacture of CAR-modified T cells to treat patients with cancers were presented. [Press release from ZIOPHARM Oncology, Inc. discussing research presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

City of Hope Physicians Present New CAR-T Cell Therapy Study Results and Other Research

Research about clinical trials that lay the groundwork for novel non-Hodgkin lymphoma, acute myeloid leukemia and two rare blood cancers, myelofibrosis and blastic plasmacytoid dendritic cell neoplasm, will be among the highlights of an upcoming meeting. [Press release from City of Hope discussing research to be presented at the 2017 American Society of Hematology (ASH) Annual Meeting, Atlanta] Press Release

Gilead Sciences and Kite to Acquire Cell Design Labs

Gilead Sciences, Inc. and its cell therapy subsidiary Kite announced that they have entered into a definitive agreement under which they have agreed to acquire Cell Design Labs, Inc., gaining new technology platforms that will enhance research and development efforts in cellular therapy. [Gilead Sciences, Inc.] Press Release | Editorial

bluebird bio and TC BioPharm Announce Strategic Collaboration to Research and Develop Gamma Delta CAR T Cell Product Candidates for Cancer Immunotherapy

bluebird bio, Inc. and TC BioPharm, Ltd. announced a strategic collaboration and license agreement focused on gamma delta CAR T cells. The companies will work together to advance TC BioPharm’s lead CAR-engineered gamma delta T cell program into clinical trials as well as on additional hematologic and solid tumor targets. [bluebird bio, Inc.] Press Release

Medigene Collaborates with RXi Pharmaceuticals to Further Sharpen Its Therapeutic T Cells

Medigene AG announced that it has entered into a research collaboration with RXi Pharmaceuticals Corporation, MA, USA to explore potential synergies of using RXi’s self-delivering RNAi technology in combination with Medigene’s recombinant TCRs to develop modified T cells with enhanced efficacy and/or safety. [Medigene AG] Press Release

New England Biolabs^® and CasZyme Collaborate to Enable Genome Editing Applications with Novel Cas9 Enzymes

New England Biolabs, Inc. and CasZyme announced that they have entered into a multi-year collaboration to identify and commercialize CRISPR-Cas nucleases, with the goal of providing researchers with additional tools for genome editing and molecular biology applications. [New England Biolabs, Inc. (PR Newswire Association LLC.)] Press Release

CGT Catapult to Collaborate with Oxford MEStar and AK (Suzhou) Biomedical as Part of Jiangsu-UK Industrial Challenge Program

The Cell and Gene Therapy (CGT) Catapult, in collaboration with Oxford MEStar and AK (Suzhou) Biomedical, announced that they have been awarded a grant from the Innovate UK-Jiangsu Industrial Challenge Program, a project set up by Innovate UK and Jiangsu Science and Technology Department to stimulate economic growth, and foster ties between China and the UK. [The Cell and Gene Therapy Catapult] Press Release

CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-Thalassemia

CRISPR Therapeutics announced the submission of a Clinical Trial Application for CTX001 in β-thalassemia. CTX001 is an investigational CRISPR gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease. [CRISPR Therapeutics] Press Release | Editorial

Juno Therapeutics Signs Licensing Agreements with Lilly, OncoTracker, and Fred Hutchinson Cancer Research Center to Advance Its BCMA-Directed Engineered T Cell Program in Multiple Myeloma with Gamma Secretase Inhibition

Juno Therapeutics, Inc. announced three license agreements to advance its program in multiple myeloma using gamma secretase inhibitors in combination with BCMA-directed CAR T cells. [Juno Therapeutics, Inc.] Press Release

ERS Genomics Licenses CRISPR/Cas9 Genome Editing Patents to Charles River

ERS Genomics announced a patent license agreement whereby U.S.-based Charles River Laboratories International, Inc. gains non-exclusive access to the company’s CRISPR/Cas9 patents to offer customers an expanded range of in vivo and in vitro genome editing services. [ERS Genomics Limited] Press Release

Mesoblast Named Global Technology Leader in Cell Therapy Industry

Mesoblast Limited was named by Frost & Sullivan as the 2017 Global Technology Leader in the Cell Therapy Industry. [Mesoblast Limited (Business Wire, Inc.)] Press Release

Nine Researchers Sue University of Rochester over Sexual-Harassment Allegations

Nine researchers filed a federal lawsuit against the University of Rochester in New York, its president Joel Seligman and its provost Robert Clark over their handling of alleged sexual harassment by a professor there. [Nature News] Editorial

Brexit ‘Breakthrough’ Raises Hope — and Questions

British science is expected to benefit from a deal that would allow EU nationals to remain in the country once it leaves the union in March 2019. [Nature News] Editorial

CAR-T Cells: The Narrow Path between Hope and Bankruptcy?

The announcement that the FDA approved Kymriah, an autologous CAR T-cell suspension targeting CD19 malignancies, raises tremendous hopes as well as uncertainties in patients and the medical community. Tisagenlecleucel is the first of its kind to reach the market, although the European Medicines Agency and FDA have approved other types of human cell-based therapies in the past. As most previously authorized cell and gene therapies were met with little commercial success thus far, all stakeholders will scrutinize the fate of the newcomer. [Bone Marrow Transplantation] Editorial

EMA

First Guidance on Monoclonal Antibodies for Use in Animals Notice

FDA

Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model; Draft Guidance for Industry; Availability (FR Doc. No:2017-26357) Notice

TGA

Varying Biological Entries on the ARTG Notice

NEW International Society for Cellular Therapy (ISCT) 2018
May 2-5, 2018
Montreal, Quebec, Canada

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

NEW Scientist – Gene Therapy for Glioblastoma (Scuola Internazionale Superiore di Studi Avanzati)

Research Technologist – Immunotherapy (STEMCELL Technologies Inc.)

Scientist – Gene Therapy (Medicines and Healthcare Products Regulatory Agency)

Postdoctoral Positions – Immunotherapy (University of Houston)

Research Scientist – Cellular Immunotherapy (Columbia Center for Translational Immunology)

Assistant Scientist – Cancer Therapy Research (University of Miami)

Postgraduate Positions – Translational Medicine (Qingdao University)

Postdoctoral Position – Cancer Research (Helmholtz Zentrum München)

Scientist – Immuno-Oncology (Regeneron Pharmaceuticals, Inc.)

Postdoctoral Fellows – ImmunoGenomics (San Raffaele-Telethon Institute for Gene Therapy)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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