Volume 17.10 | Mar 14

Issue 17.10 March 14, 2016
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Stem Cells Regenerate Human Lens after Cataract Surgery, Restoring Vision
Researchers have developed a new, regenerative medicine approach to remove congenital cataracts in infants, permitting remaining stem cells to regrow functional lenses. The treatment, which has been tested in animals and in a small, human clinical trial, produced much fewer surgical complications than the current standard-of-care and resulted in regenerated lenses with superior visual function in all 12 of the pediatric cataract patients who received the new surgery. [Press release from the University of California, San Diego discussing online prepublication in Nature] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Targeted Gene Addition in Human CD34+ Hematopoietic Cells for Correction of X-Linked Chronic Granulomatous Disease
Scientists demonstrated that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human hematopoietic stem and progenitor cells approaches clinically relevant levels of targeted integration into the AAVS1 safe harbor locus. [Nat Biotechnol] Abstract

Biodegradable Scaffold with Built-In Vasculature for Organ-on-a-Chip Engineering and Direct Surgical Anastomosis
Investigators report the fabrication of a scaffold that supports the assembly of parenchymal cells on a mechanically tunable matrix surrounding a perfusable, branched, three-dimensional microchannel network coated with endothelial cells. [Nat Mater] Abstract | Press Release

Evidence for Mechanisms Underlying the Functional Benefits of a Myocardial Matrix Hydrogel for Post-MI Treatment
An injectable hydrogel derived from decellularized porcine ventricular myocardium has been shown to halt the post-infarction progression of negative left ventricular remodeling and decline in cardiac function in both small and large animal models. Researchers sought to elucidate the tissue-level mechanisms underlying the therapeutic benefits of myocardial matrix injection. [J Am Coll Cardiol] Abstract | Press Release | Video | Podcast

Antigen-Encoding Bone Marrow Terminates Islet-Directed Memory CD8+ T-Cell Responses to Alleviate Islet Transplant Rejection
Scientists showed that transfer of bone marrow encoding cognate antigen directed to dendritic cells, under mild, immune-preserving conditions inactivates established memory CD8+ T-cell populations and generates a long-lived, antigen-specific tolerogenic environment. [Diabetes] Abstract

Colored Cornea Replacements with Anti-Infective Properties: Expanding the Safe Use of Silver Nanoparticles in Regenerative Medicine
Investigators report the development of a new strategy to pre-stabilize and incorporate silver nanoparticles with different colors into collagen matrices for fabrication of corneal implants and lenses, and assessed their in vitro and in vivo activity. [Nanoscale] Abstract

Systemic Delivery of chTNT-3/CpG Immunoconjugates for Immunotherapy in Murine Solid Tumor Models
CpG oligodeoxynucleotides (CpG) chemically conjugated to chTNT-3 (chTNT-3/CpG) were compared to free CpG in their ability to stimulate the immune system in vitro and reduce tumor burden in vivo. [Cancer Immunol Immunother] Abstract

Comparing the Effects of MSCs and CD34+ Cell Therapy in a Rat Model of Myocardial Infarction
Scientists compared human umbilical cord blood (HUCB)-derived CD34+ and HUCB-derived MSCs for the repair of cardiac tissue by induction of the angiogenesis. [IUBMB Life] Abstract

A Specific Aptamer-Cell Penetrating Peptides Complex Delivered siRNA Efficiently and Suppressed Prostate Tumor Growth In Vivo
Investigators aimed to address both specificity and efficiency of SURVIVIN-siRNA delivery by constructing a therapeutic complex using combinatorial strategies. A fusion protein STD was first expressed to serve as a backbone, consisting of streptavidin, a cell-penetrating peptide called trans-activator of transcription and a double-stranded RNA binding domain. [Cancer Biol Ther] Abstract

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Cell Therapy in Chronic Liver Disease
Mesenchymal stem cells are promising candidates for cell transplantation, as they have been shown to reduce liver fibrosis and support endogenous regeneration. Adipose tissue-derived stem cells are also being tested in this setting, because of their ready availability. [Curr Opin Gastroenterol] Abstract

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

Free Wallchart from Nature Reviews Neuroscience on Modeling Neurological Disease with iPS Cells
Benitec’s Hepatitis B Therapy Reduces HBV DNA by 98.5% In Vivo
Benitec Biopharma Limited announced that BB-HB-331, a DNA-directed RNA interference therapy targeting the hepatitis B virus (HBV), demonstrates robust and durable suppression of HBV in vivo following a single administration. [Press release from the Benitec Biopharma Limited discussing research to be presented at the HEP DART 2015, Wailea] Press Release

TiGenix Announces Positive 52-Week Phase III Results of Cx601 in Complex Perianal Fistulas in Crohn’s Disease Patients
TiGenix NV announced that a single injection of Cx601 was statistically superior to placebo in achieving combined remission at week 52 in the Phase III ADMIRE-CD trial in Crohn’s disease patients with complex perianal fistulas with inadequate response to previous therapies, including anti-TNFs. [TiGenix NV (GlobeNewswire, Inc.)] Press Release

Atreca Announces Presentation of Preclinical Data for Cancer Immunotherapy Program
Atreca, Inc. announced positive preclinical findings generated using the company’s Immune Repertoire Capture™ technology. Immune Repertoire Capture™ technology generated the sequences of native antibodies from the active immune response of an individual with Stage 4 lung adenocarcinoma who had experienced long-term non-progression of disease. [Press release from the Atreca, Inc. discussing research to be presented at the Keystone Symposia Conference “Antibodies as Drugs”, Whistler] Press Release

Combination of Lapatinib and Trastuzumab Shrinks HER2 Positive Breast Cancer Significantly in 11 Days after Diagnosis
Approximately a quarter of women with HER2 positive breast cancer, who were treated with a combination of the targeted drugs lapatinib and trastuzumab before surgery and chemotherapy, saw their tumors shrink significantly or even disappear, according to results from a clinical trial. [Press release from The University of Manchester discussing research presented at 10th European Breast Cancer Conference (EBCC-10), Amsterdam] Press Release

Biothera Pharmaceuticals to Present Its Phase II Cancer Immunotherapy Data
Biothera Pharmaceuticals Inc. presented data on its Phase II cancer immunotherapy Imprime PGG, the company’s first-in-class, systemically administered beta glucan pathogen associated molecular patterning molecule. [Press release from Biothera Pharmaceuticals Inc. discussing research presented at the Molecular Medicine Tri-Conference 2016, San Francisco] Press Release

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Accusations of Errors and Deception Fly in CRISPR Patent Fight
Adversaries in the legal battle over the rights to the CRISPR gene-editing technology are preparing to fire their initial shots. In two documents filed with the U.S. Patent Trial and Appeal Board, lawyers for the Regents of the University of California and the Broad Institute, offered hints at how they will lay claim to the breakthrough technology and its financial spoils. [ScienceInsider] Editorial

Statisticians Issue Warning over Misuse of P Values
Misuse of the P value is contributing to the number of research findings that cannot be reproduced, the American Statistical Association warns in a statement. The group has taken the unusual step of issuing principles to guide use of the P value, which it says cannot determine whether a hypothesis is true or whether results are important. [Nature News] Editorial
Baylor College of Medicine, Baylor Scott & White Health Announce Collaboration on Biomedical Research
Baylor College of Medicine and Baylor Scott & White Health have entered into an agreement to expand biomedical research in North and Central Texas. [Baylor Scott & White Health] Press Release

Temple University Hospital Offering New Trial Using Donor Stem Cells to Treat Heart Failure
Temple University Hospital is participating in a nationwide, Phase III clinical trial investigating stem cells as a treatment for patients with chronic heart failure due to left ventricular systolic dysfunction. The trial, dubbed DREAM-HF, is a double-blind, randomized, placebo controlled trial investigating whether donor stem cells which are injected into the damaged portion of the heart can restore heart muscle function. [Temple University] Press Release

Data Safety and Monitoring Board Recommends Celldex’s Phase III Study of RINTEGA® (Rindopepimut) in Newly Diagnosed Glioblastoma Be Discontinued as It Is Unlikely to Meet Primary Overall Survival Endpoint in Patients with Minimal Residual Disease
Celldex Therapeutics, Inc. announced that the independent Data Safety and Monitoring Board has determined, based on a preplanned interim analysis, that continuation of the Phase III ACT IV study of RINTEGA® in patients with newly diagnosed EGFRvIII-positive glioblastoma will not reach statistical significance for overall survival in patients with minimal residual disease, the primary endpoint of the study, as both the RINTEGA arm and the control arm are performing on par with each other. [Celldex Therapeutics, Inc.] Press Release

Vericel Announces Positive Top-Line Results from Phase IIb ixCELL-DCM Clinical Trial of Ixmyelocel-T in Patients with Heart Failure Due to Ischemic Dilated Cardiomyopathy
Vericel Corporation announced top-line results from the company’s Phase IIb ixCELL-DCM clinical trial of ixmyelocel-T in patients with advanced heart failure due to ischemic dilated cardiomyopathy (DCM). [Vericel Corporation] Press Release

Data and Safety Monitoring Board Recommends Advance of Current DC Vaccine Clinical Trial in AML into Phase II
Medigene AG announced the successful recruitment of the planned patients for Phase I of its current clinical trial of dendritic cell (DC) vaccines in acute myeloid leukemia (AML). [Medigene AG] Press Release

Alliance for Cancer Gene Therapy Announces Three New Grant Awardees for Cancer Research
Alliance for Cancer Gene Therapy (ACGT) announced the recipients of its 2016 ACGT Young Investigator Grants. These grants are awarded to help the researchers build upon earlier research and take them to the next level in cancer treatment, setting the stage for possible clinical trials. [Alliance for Cancer Gene Therapy (EurekAlert)] Press Release

Spark Therapeutics Announces Acquisition of Genable Technologies
Spark Therapeutics announced that it has acquired Genable Technologies, a private gene therapy innovator with which Spark has collaborated since 2014 in the development of Genable’s therapeutic program targeting one of the most prevalent forms of inherited retinal disease. [Spark Therapeutics, Inc.] Press Release

Peregrine Pharmaceuticals Announces Formal Commissioning of New, State-of-the-Art Commercial Manufacturing Facility
Peregrine Pharmaceuticals, Inc. announced that its new state-of-the-art commercial biomanufacturing suite in Tustin, California, has been formally commissioned. As part of the commissioning process, all relevant regulatory agencies have been notified and cGMP production is currently underway. [Peregrine Pharmaceuticals, Inc.] Press Release

Celsion Corporation Announces Launch of the OPTIMA Study in China
Celsion Corporation announced the launch of the OPTIMA Study in China. The OPTIMA Study is the company’s global pivotal, double-blind, placebo-controlled trial, evaluating ThermoDox®, Celsion’s proprietary heat-activated liposomal encapsulation of doxorubicin, in combination with radiofrequency ablation standardized to 45 minutes (sRFA) versus sRFA alone to treat newly diagnosed patients with hepatocellular carcinoma. [Celsion Corporation] Press Release
National Cancer Institute; Notice of Meeting (FR Doc. No: 2016-05334)
Investigating and Reporting Adverse Reactions Related to Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps) Regulated Solely under Section 361 of the Public Health Service Act and 21 CFR Part 1271 – Guidance for Industry
Food and Drug Administration (United States)

Removal of Review and Reclassification Procedures for Biological Products Licensed Prior to July 1, 1972 (FR Doc. No: 2016-02884)
NEW Advances in Immuno-Oncology Congress 2016
May 12-13, 2016
London, United Kingdom

NEW 14th International Cord Blood Symposium
June 9-11, 2016
San Francisco, United States

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

NEW Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

Scientist – Human Pluripotent Stem Cell Neural Differentiation (STEMCELL Technologies Inc.)

Postdoctoral Fellowship – Adeno-Associated Virus Gene Therapy (Icahn School of Medicine at Mount Sinai)

Postdoctoral Fellow – Hematology/Oncology (Boston Children’s Hospital)

Postdoctoral Fellowship – Stem Cells, Oligodendrogenesis and Remyelination (UC Davis)

PhD Positions – Cancer, Microbiology and Stem Cell Engineering (Virginia Commonwealth University)

PhD Positions – Tendon Repair and Regeneration (NUI Galway)

PhD Position – Leukemogenesis and Treatment Resistance (Institute for Tumor Biology and Experimental Therapy)

Postdoctoral Positions – Gene Regulation and Translational Cancer Research (University of Texas Health Science Center)

Postdoctoral Position – Hematology/Oncology (Penn State College of Medicine)

Postdoctoral Fellow/Research Associate – Cancer Therapy (Memorial Sloan Kettering Cancer Center)

Translational Research Scientist – Hematology/Oncology (University of Chicago)

Staff Scientist – Cell Therapy Process Development (Fred Hutchinson Cancer Research Center)

Postdoctoral Research Fellow – Cell Cycle Control and Tumorigenesis (Fred Hutchinson Cancer Research Center)

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