Volume 16.06 | Feb 16

Newsletter Issue
Issue 16.06 February 16, 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
Size of Biomarker in Transplanted Blood Cells Associated with Improved Survival following Transplantation
Among patients with severe aplastic anemia who received stem cell transplant from an unrelated donor, longer leukocyte telomere length was associated with increased overall survival at five years. [Press release from The Journal of the American Medical Association (JAMA)] Press Release | Editorial | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Off-Target-Free Gene Delivery by Affinity-Purified Receptor-Targeted Viral Vectors
Investigators described receptor-targeted adeno-associated viral vectors that allow genetic modification of rare cell types ex vivo and in vivo while showing no detectable off-targeting. [Nat Commun] Abstract | Press Release

Transplantation of Bovine Adrenocortical Cells Encapsulated in Alginate
Scientists created a bioartificial adrenal with 3D cell culture conditions by encapsulation of bovine adrenocortical cells in alginate (enBACs). The enBACs were transplanted into adrenalectomized, immunodeficient, and immunocompetent rats. [Proc Natl Acad Sci USA] Abstract | Full Article | Press Release

Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression
Researchers investigated the feasibility and safety of triggering proliferation in vivo by temporary suppression of the cyclin-dependent kinase inhibitor, p21. AAV-mediated, acute knockdown of p21 in intact skeletal muscles elicited proliferation of multiple, otherwise quiescent cell types, notably including satellite cells. [Mol Ther] Abstract

Blockade of CD40-CD154 Costimulatory Pathway Promotes Long-Term Survival of Full-Thickness Porcine Corneal Grafts in Nonhuman Primates: Clinically Applicable Xenocorneal Transplantation
Scientists investigated the feasibility of full-thickness porcine corneas as human corneal substitutes using a CD40-CD154 costimulatory pathway blocking strategy in a clinically applicable pig-to-nonhuman primate corneal transplantation model. [Am J Transplant] Abstract

Brentuximab Vedotin in Patients with Hodgkin Lymphoma and a Failed Allogeneic Stem Cell Transplantation: Results from a Named Patient Program at Four Italian Centers
Researchers retrospectively evaluated the efficacy and safety of brentuximab vedotin in a multicenter setting of classical Hodgkin lymphoma relapsing or progressing after allogeneic stem cell transplantation. [Oncologist] Abstract

The Safety of Allogeneic Innate Lymphocyte Therapy for Glioma Patients with Prior Cranial Irradiation
Investigators evaluated γδ T cell toxicity by assessing actual and correlative toxicity indicators in several available models including microglial activation and expression of stress-induced ligands in mouse brain after whole-brain irradiation. [Cancer Immunol Immunother] Abstract

Human Placenta-Derived Adherent Cells Improve Cardiac Performance in Mice with Chronic Heart Failure
Researchers tested the therapeutic effects of PDA-001, an intravenous formulation of placenta-derived adherent cells, in mice with chronic heart failure (CHF). Three weeks after transaortic constriction surgery to induce CHF, the mice underwent direct intramyocardial or i.v. injection of PDA-001 at a high, medium, or low dose. [Stem Cells Transl Med] Abstract

Long-Term In Vivo Gene Expression in Mouse Kidney Using φC31 Integrase and Electroporation
Researchers developed an in vivo long-term gene expression system in murine kidney using φC31 integrase. The long-term in vivo gene expression system in the kidney was achieved by co-transfecting 5 µg of pORF-luc/attB as a donor plasmid and 20 µg of pCMV-luc as a helper plasmid into the right kidney of mice by electroporation. [J Drug Target] Abstract

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REVIEWS
New Strategies in Glioblastoma: Exploiting the New Biology
The last five years has seen an explosion in our understanding of the genetic and molecular underpinnings of glioblastoma tumors leading to renewed optimism about potential new therapeutic approaches. The author reviews the excitement and challenges that face the development of effective new treatments that exploit this new biology. [Clin Cancer Res] Abstract

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs
The authors compared the preclinical studies of adeno-associated virus vectors used to treat dogs with hemophilia B with the results obtained in subsequent human clinical trials using muscle- and liver-based approaches. [Hum Gene Ther Clin Dev] Abstract

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

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SCIENCE NEWS
Baxter Provides Progress Update on Gene Therapy Program, Including Phase I/II Clinical Trial of BAX 335, Investigational Gene Therapy for Hemophilia B
Baxter International Inc. provided an update on its gene therapy program, including progress on the Phase I/II open-label clinical trial assessing the safety and optimal dosing level of BAX 335, an investigational factor IX gene therapy treatment for hemophilia B. [Press release from Baxter International Inc. discussing research presented at the 8th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Helsinki] Press Release

Biothera Immunotherapy Binds to Multiple Immune Cell Receptors to Kill Cancer
Biothera’s late stage cancer immunotherapy drug binds to multiple innate immune cell receptors to exert enhanced cytotoxicity against tumor cells. Researchers presented new data showing Imprime PGG® binds to Fc gamma receptors in addition to complement receptor 3 on human neutrophils and monocytes. [Press release from Biothera discussing research presented at the Keystone Symposium on Tumor Immunology, Banff] Press Release | Poster

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POLICY
Open Records Laws Becoming Vehicle for Harassing Academic Researchers, Report Warns
The electronic age of communication is making it easier for activists, companies and lobbying groups to use state open records laws — designed to promote transparency — to harass academic researchers they disagree with, a scientific-integrity group warns in a new report. [ScienceInsider] Editorial | Report

Stem Cell Pioneer Joins Forces with Stem Cell Fraudster
The scientist who once fraudulently claimed to have created embryonic stem cells matched to human patients and the one who really did it plan to conduct joint research. [ScienceInsider] Editorial
 
BUSINESS
LYSOGENE, University of Massachusetts Medical School and Auburn University Announce Collaboration Agreement
LYSOGENE announced that it has entered into a strategic collaboration with the University of Massachusetts Medical School (UMMS) and Auburn University (AU). Through the collaboration, LYSOGENE, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus gene therapy technology. [LYSOGENE]
Press Release

Advaxis Forms Clinical Trial Collaboration with Incyte to Evaluate Investigational Combination of Two Novel Cancer Immunotherapies for Early Stage Cervical Cancer
Advaxis, Inc. has entered into a clinical trial collaboration agreement with Incyte Corporation to evaluate the combination of Advaxis’s Lm-LLO cancer immunotherapy, ADXS-HPV, with Incyte’s investigational oral indoleamine 2,3-dioxygenase 1 inhibitor, epacadostat. [Advaxis, Inc.] Press Release

TiGenix and Lonza Sign Agreement for the Manufacture of Stem Cell-Based Treatment
Lonza and TiGenix announced an agreement for the supply of TiGenix’s expanded adipose-derived stem cell product, Cx601. Under the agreement, Lonza will manufacture material for the Phase III trial of Cx601 in the US at Lonza’s cell therapy production facility in Walkersville, Maryland. [Lonza Group Ltd.] Press Release

Voyager Therapeutics & Genzyme Announce Major Strategic Collaboration to Develop and Commercialize Novel AAV Gene Therapies for Patients with CNS Disorders
Voyager Therapeutics and Genzyme announced a major strategic collaboration to discover, develop and commercialize novel gene therapies for severe central nervous system (CNS) disorders. [Genzyme Corporation] Press Release

Panacea Pharmaceuticals Engages Accelovance for Clinical Development of Its Novel Nanoparticle-Based Therapeutic Cancer Vaccine
Panacea Pharmaceuticals, Inc. and Accelovance Inc. announced the establishment of a clinical development services agreement to develop and advance Panacea’s oncology immunotherapy pipeline. [Panacea Pharmaceuticals, Inc. (PR Newswire Association LLC)] Press Release

Manufacturing Authorization of Gene Therapy Medicinal Products Granted to CELLforCURE, the LFB Group Subsidiary Specialized in Innovative Therapies
CELLforCURE was granted a new manufacturing authorization. Delivered by the French national agency for medicines and health products safety, this authorization allows now CELLforCURE to manufacture gene therapy drugs. [LFB Group] Press Release

Enrollment Begins European Knee Meniscus Injury Pilot Trial to Evaluate Cytori Cell Therapy
Cytori Therapeutics, Inc. announced that the first patient was enrolled and treated this week in a single arm, open label investigator initiated trial of Cytori Cell Therapy in patients undergoing surgery for traumatic meniscal injuries in the knee. [Cytori Therapeutics, Inc.] Press Release

Amgen Announces FDA Advisory Committees to Review Talimogene Laherparepvec for the Treatment of Patients with Metastatic Melanoma
Amgen announced that the Cellular, Tissue and Gene Therapies Advisory Committee and the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration will jointly review the company’s Biologics License Application for talimogene laherparepvec. [Amgen Inc.] Press Release
 
NIH
Announcement of a Draft NIH Policy on Dissemination of NIH-Funded Clinical Trial Information (FR Doc. No: 2015-02994)

Clinical Trials Registration and Results Submission (FR Doc. No: 2015-02990)
 
CBER
Cellular & Gene Therapy Guidances
 
REGULATORY
Food and Drug Administration (United States)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Export of Food and Drug Administration Regulated Products: Export Certificates; Correction (FR Doc. No: 2015-03005)

Joint Meeting of the Cellular, Tissue and Gene Therapies Advisory Committee and the Oncologic Drug Advisory Committee; Notice of Meeting (FR Doc. No: 2015-02910)

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Food and Drug Administration Recall Regulations (FR Doc. No: 2015-02788)

Brief Summary and Adequate Directions for Use: Disclosing Risk Information in Consumer-Directed Print Advertisements and Promotional Labeling for Human Prescription Drugs; Revised Draft Guidance for Industry; Availability (FR Doc. No: 2015-02527)
 
EVENTS
NEW South Central Association of Blood Banks (SCABB) Mid-Atlantic Symposium: In Search of the Safe & Effective Transfusion
April 10, 2015
Annapolis, United States

NEW The Biology of Regenerative Medicines
April 22-24, 2015
Cambridge, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
 
JOB OPPORTUNITIES
NEW Research Technician in AAV Gene Therapy (Icahn School of Medicine at Mount Sinai)

NEW T Cell Process Development Scientist/Senior Scientist (Adaptimmune Ltd.)

NEW Postdoctoral Associate – Developing Adeno-Associated (AAV) and Non-Virus Vectors for Treating Monogenic Disorders (University of Massachusetts)

Research Associate – Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

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

Scientist – PSC Biology and Bioengineering (STEMCELL Technologies Inc.)

Scientist – Pluripotent Stem Cell Media Development, High Throughput Screening (STEMCELL Technologies Inc.)

Scientist – Cell Culture Support Products (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Senior Process Development Engineer (Sangamo BioSciences, Inc.)

Director – Vector Production (Sangamo BioSciences, Inc.)

Postdoctoral Fellow – Vascular Bioengineering (University of Pittsburgh)

Assistant/Associate Professor – Gastroenterology (North Carolina State University College of Veterinary Medicine)

Postdoctoral Fellow – Wnt Signaling in Development and Disease (Van Andel Research Institute)

Postdoctoral Fellowship – CNS Inflammation (Rutgers University)


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