Volume 16.16 | Apr 27

Newsletter Issue
Issue 16.16 April 27, 2015
Cell Therapy News - Your Industry in an Instant
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Publications | Reviews | Science | Policy | Business | NIH | CBER | Regulatory | Events | Jobs
 
TOP STORY
Researchers Get under the Skin to Develop New Transplant Technique
Researchers describe developing a new site for islet transplantation under the skin, which they believe will offer less risk and far greater health benefits for patients. Islet transplantation is a procedure that temporarily allows people with severe diabetes to stop taking insulin. [Press release from University of Alberta discussing online publication in Nature Biotechnology] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Mutant MHC Class II Epitopes Drive Therapeutic Immune Responses to Cancer
Recently, the authors proposed a personalized immunotherapy approach to target the full spectrum of a patient’s individual tumor-specific mutations. They showed in three independent murine tumor models that a considerable fraction of non-synonymous cancer mutations is immunogenic and that, unexpectedly, the majority of the immunogenic mutanome is recognized by CD4+ T cells. [Nature] Abstract | Press Release

Selective Elimination of Mitochondrial Mutations in the Germline by Genome Editing
Researchers report on a strategy toward preventing germline transmission of mitochondrial diseases by inducing mtDNA heteroplasmy shift through the selective elimination of mutated mtDNA. As a proof of concept, investigators took advantage of NZB/BALB heteroplasmic mice, which contain two mtDNA haplotypes, BALB and NZB, and selectively prevented their germline transmission using either mitochondria-targeted restriction endonucleases or TALENs. [Cell] Abstract | Graphical Abstract | Press Release

Outcomes following Gene Therapy in Patients with Severe Wiskott-Aldrich Syndrome
Investigators assessed the outcomes and safety of autologous hematopoietic stem cell (HSC) gene therapy in Wiskott-Aldrich syndrome. Gene-corrected autologous HSCs were infused in seven consecutive patients with severe Wiskott-Aldrich syndrome lacking HLA antigen-matched related or unrelated HSC donors following myeloablative conditioning. [JAMA] Abstract | Press Release

Monoclonal Antibodies against GARP/TGF-β1 Complexes Inhibit the Immunosuppressive Activity of Human Regulatory T Cells In Vivo
Two anti-GARP monoclonal antibodies were generated that block the production of active transforming growth factor–β1 (TGF-β1) by human regulatory T cells (Tregs). In a model of xenogeneic graft-versus-host disease in NSG mice, the blocking antibodies inhibited the immunosuppressive activity of human Tregs. [Sci Transl Med] Abstract | Press Release

Targeting Breast to Brain Metastatic Tumors with Death Receptor Ligand Expressing Therapeutic Stem Cells
Investigators explored the ability of engineered adult stem cells to track metastatic deposits in this model and showed that engineered stem cells either implanted or injected via circulation efficiently home to metastatic tumor deposits in the brain. [Brain] Abstract | Press Release

XactMice: Humanizing Mouse Bone Marrow Enables Microenvironment Reconstitution in a Patient-Derived Xenograft Model of Head and Neck Cancer
Researchers developed a technique to expand human hematopoietic stem and progenitor cells and used them to reconstitute the radiation-depleted bone marrow of a NOD/SCID/IL2rg-/- mouse on which a patient’s tumor is then transplanted (XactMice). [Oncogene] Abstract | Press Release

CNTF Gene Therapy Confers Lifelong Neuroprotection in a Mouse Model of Human Retinitis Pigmentosa
The long-term outcome of neuroprotection as a therapeutic strategy for preventing cell death in neurodegenerative disorders remains unknown, primarily due to slow disease progression and the inherent difficulty of assessing neuronal survival in vivo. Employing a murine model of retinal disease, scientists demonstrated that ciliary neurotrophic factor (CNTF) confers life-long protection against photoreceptor degeneration. [Mol Ther] Abstract

Lentivector Knock-Down of CCR5 in Hematopoietic Stem Cells Confers Functional and Persistent HIV-1 Resistance in Humanized Mice
Investigators combined CCR5 knock-down by a highly efficient miRNA lentivector with pre-transplantation selection of transduced hematopoietic stem and progenitor cells to obtain a rather pure population of gene engineered CD34+ cells. [J Virol] Abstract

Placental Mesenchymal Stromal Cells Rescue Ambulation in Ovine Myelomeningocele
Scientists demonstrated that using early gestation human placenta-derived mesenchymal stromal cells to augment in utero repair of myelomeningocele (MMC) resulted in significant and consistent improvement in neurologic function at birth in the rigorous fetal ovine model of MMC. [Stem Cells Transl Med]
Abstract | Press Release | Video

CRISPR/Cas9-Mediated Gene Editing in Human Tripronuclear Zygotes
Investigators used tripronuclear zygotes to further investigate clustered regularly interspaced short palindromic repeat-associated system (CRSIPR/Cas9)-mediated gene editing in human cells. They found that CRISPR/Cas9 could effectively cleave the endogenous β-globin gene. [Protein Cell] Full Article | Editorial

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REVIEWS
The Endometrium as a Source of Mesenchymal Stem Cells for Regenerative Medicine
The endometrium has emerged as an attractive novel source of adult stem cells that are easily accessed and demonstrate remarkable differentiation capacity. The authors summarize the current understanding of endometrial stem cells and their therapeutic potential in regenerative medicine. [Biol Reprod] Abstract | Full Article

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research fields.

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SCIENCE NEWS
Transgene Presents New Data with TG1050, an Immunotherapy Being Developed to Treat Chronic Hepatitis B
Transgene SA announced that new pre-clinical data with TG1050, an immunotherapy being developed for the treatment of chronic hepatitis B. The data presented demonstrate the antiviral potential of TG1050 in a persistent hepatitis B virus model. [Press release from Transgene SA discussing research presented at The International Liver Congress™ 2015, Vienna] Press Release

Alnylam Reports 12-Month Clinical Data from Phase II Open Label Extension (OLE) Study of Patisiran, an Investigational RNAi Therapeutic for Patients with Familial Amyloidotic Polyneuropathy (FAP)
Alnylam Pharmaceuticals, Inc. announced initial 12-month clinical data from its ongoing Phase II OLE study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated amyloidosis in patients with FAP. [Press release Alnylam Pharmaceuticals, Inc. discussing research presented at the 67th Annual Meeting of the American Academy of Neurology (AAN), Washington D.C.] Press Release

SillaJen Presents Data Demonstrating Pexa-Vec Synergy with Angiogenesis Inhibitors: A Collaboration with University of California, San Francisco
SillaJen, Inc. announced an oral and poster presentation of research demonstrating the synergy of a murine adapted version of its lead product candidate, Pexa-Vec with angiogenesis inhibitors. [Press release from SillaJen, Inc. (PR Newswire Association LLC) discussing research to be presented at the 2015 Annual Meeting of the American Association for Cancer Research (AACR), Philadelphia] Press Release

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POLICY
High-Scoring Grant Applications Yield More Highly Cited Papers
Grant applications that are ranked more highly by peer reviewers go on to yield more patents and highly cited papers, an analysis of more than 130,000 funded grants finds. The study contradicts the widely held belief — supported by some previous findings — that there is scant connection between reviewers’ scores of a research proposal and its subsequent impact. [Nature News] Editorial | Abstract

Canadian Budget Pushes Applied Research
The reigning Conservative party has introduced a budget that emphasizes applied research and scientific collaboration with industry. The Canada Foundation for Innovation would receive $1.33 billion in new money for university and hospital research facilities, to be doled out over six years beginning in 2017. [Nature News] Editorial
 
BUSINESS
AstraZeneca Enters Strategic Immuno-Oncology Collaboration with Celgene Corporation to Develop PD-L1 Inhibitor Program for Patients with Serious Blood Cancers
AstraZeneca and MedImmune announced that they have entered into an exclusive collaboration agreement with Celgene Corporation, a global leader in hematological cancers, for the development and commercialization of MEDI4736 across a range of blood cancers including non-Hodgkin’s lymphoma, myelodysplastic syndromes and multiple myeloma. [AstraZeneca] Press Release

AstraZeneca and Innate Pharma Announce Global Co-Development and Commercialization Collaboration for IPH2201 in Immuno-Oncology
AstraZeneca and MedImmune announced that they have entered into a collaboration to accelerate and broaden the development of Innate Pharma SA’s proprietary anti-NKG2A antibody, IPH2201, including in combination with MEDI4736, an anti-PD-L1 immune checkpoint inhibitor developed by MedImmune. [AstraZeneca] Press Release

MedImmune and Juno Therapeutics Announce Immuno-Oncology Clinical Trial Collaboration
MedImmune and Juno Therapeutics, Inc. announced that they have entered into a new collaboration to conduct combination clinical trials in immuno-oncology with one of Juno’s investigational CD19-directed chimeric antigen receptor T cell candidates and MedImmune’s investigational programmed cell death ligand 1 immune checkpoint inhibitor, MEDI4736. [MedImmune, LLC] Press Release

MaxCyte and Johns Hopkins University Announce Strategic Immuno-Oncology Collaboration to Advance CAR T-Cell Therapies
MaxCyte® Inc. announced a strategic research collaboration with Johns Hopkins University to develop unique Chimeric Antigen Receptor (CAR) T-cell therapies, which harness patients’ own immune systems to combat cancers. [MaxCyte® Inc.] Press Release

Astellas Pharma and Potenza Therapeutics Partner to Build a Portfolio of Immuno-Oncology Therapeutics
Astellas Pharma Inc. and Potenza Therapeutics, Inc. announced an exclusive research and development collaboration. The goal of the collaboration is to advance a portfolio consisting of programs with novel mechanisms of action targeting immune checkpoint pathways, co-stimulatory signals and regulatory T cells. [Potenza Therapeutics, Inc.] Press Release

Global Strategic Partners Merck and Pfizer Initiate Phase III Study with Avelumab in Patients with Stage IIIb/IV Non-Small Cell Lung Cancer
Merck and Pfizer announced the initiation and first patient treated in the international Phase III study designed to assess the efficacy and safety of the investigational cancer immunotherapy avelumab, compared with docetaxel, in patients with stage IIIb/IV non-small cell lung cancer who have experienced disease progression after receiving a prior platinum-containing doublet therapy. [Pfizer Inc.] Press Release

Bristol-Myers Squibb Receives Positive CHMP Opinion in the European Union for Opdivo (Nivolumab) for the Treatment of Advanced Melanoma in Both First-Line and Previously Treated Patients
Bristol-Myers Squibb Company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending that Opdivo, a PD-1 immune checkpoint inhibitor, be granted approval for use in both first-line and previously treated patients with advanced (unresectable or metastatic) melanoma. [Bristol-Myers Squibb Company] Press Release

Celladon Reports Negative Results for CUPID2 Trial of MYDICAR® in Advanced Heart Failure
Celladon Corporation announced that its Phase IIb CUPID2 trial did not meet its primary and secondary endpoints. CUPID2 is a randomized, double-blind, placebo-controlled, multinational trial evaluating a single, one-time, intracoronary infusion of the cardiovascular gene therapy agent MYDICAR® (AAV1/SERCA2a) versus placebo added to a maximal, optimized heart failure drug and device regimen. [Celladon Corporation] Press Release

Visabl Website Update Launched
Visabl.com, the powerful cloud based antibody and biomarker search engine, announced the largest website update to date. The Visabl.com antibody search function has been completely overhauled, and has the ability to dynamically sort and populate antibodies as you type. [Visabl] Press Release
 
NIH
National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc. No: 2015-09502)
 
CBER
Approval Letter – Gardasil
 
REGULATORY
Food and Drug Administration (United States)

Determination of Regulatory Review Period for Purposes of Patent Extension; FLUBLOK (FR Doc. No: 2015-09521)

Clinical Trial Endpoints for the Approval of Non-Small Cell Lung Cancer Drugs and Biologics; Guidance for Industry; Availability (FR Doc. No: 2015-09303)
 
EVENTS
NEW Stem Cells & Regenerative Medicine Congress USA
September 2-3, 2015
Washington D.C., United States

NEW International Society for Experimental Hematology (ISEH) 44th Annual Scientific Meeting
September 17-19, 2015
Kyoto, Japan

NEW Cells: From Robert Hooke to Cell Therapy – A 350 Year Journey
October 5-6, 2015
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Research Assistant – Exosome-Based Gene Therapy for Huntington’s Disease (University of Oxford)

NEW Postdoctoral Fellow – Neural Tissue Engineering (Nanyang Technological University)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Director – Clinical Trial Operations (BioRestorative Therapies, Inc.)

Director – Cell Therapy Manufacturing (BioRestorative Therapies, Inc.)

Professor – Gene Therapy (University of Massachusetts)

Research Scientist – Cell and Gene Therapy Group (Biogen Idec)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

Postdoctoral Positions – Developmental Biology (Joslin Diabetes Center)

Cell and Gene Therapy Formulation Scientist (Stelfox)

Associate Director, Center for Gene Therapy (City of Hope)


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