Cell Therapy News Volume 18.29 | Aug 28 2017

TOP STORY

Intravesical rAd–IFNα/Syn3 for Patients with High-Grade, Bacillus Calmette-Guerin–Refractory or Relapsed Non–Muscle-Invasive Bladder Cancer: A Phase II Randomized Study

Scientists assessed the efficacy and safety of recombinant adenovirus interferon alpha with Syn3 (rAd–IFNα/Syn3), a replication-deficient recombinant adenovirus gene transfer vector, for patients with high-grade bacillus Calmette-Guerin-refractory or relapsed non–muscle-invasive bladder cancer. [J Clin Oncol] Abstract | Press Release

(Ranked by impact factor of the journal)

Phase I Clinical Trial Using mbIL21 Ex-Vivo Expanded Donor-Derived NK Cells after Haploidentical Transplantation

To test the hypothesis that NK cells can decrease the risk of leukemia relapse, investigators initiated a Phase I dose-escalation study of expanded donor NK cells infused before and after haploidentical hematopoietic stem cell transplantation for high-risk myeloid malignancies. [Blood] Abstract

Delineation of the Timing of Second Line Therapy Post-Autologous Stem Cell Transplant in Patients with AL Amyloidosis

The authors conducted a retrospective study to evaluate the patterns of relapse or progression and the timing of re-initiating therapy among 235 patients initially treated with autologous stem cell transplantation at Mayo Clinic. [Blood] Abstract

Genome Editing for Scalable Production of Alloantigen-Free Lentiviral Vectors for In Vivo Gene Therapy

Investigators showed that producer cell-derived polymorphic class-I major histocompatibility complexes are incorporated into the lentiviral vector surface and trigger allogeneic T-cell responses. [EMBO Mol Med] Full Article | Graphical Abstract

Local Gene Therapy Durably Restores Vestibular Function in a Mouse Model of Usher Syndrome Type 1G

Researchers showed that a single delivery of the sans cDNA by the adenoassociated virus 8 to the inner ear of newborn mutant mice reestablishes the expression and targeting of the protein to the tips of stereocilia. [Proc Natl Acad Sci USA] Full Article

Low Oxidative Stress-Mediated Proliferation via JNK-FOXO3a-Catalase Signaling in Transplanted Adult Stem Cells Promotes Wound Tissue Regeneration

Pre-clinically, 10µM H₂O₂-mediated pre-conditioning of bone marrow stromal cells/hematopoietic stem/progenitor cells transplantation accelerated wound closure, enhanced catalase expression and decreased reactive oxygen species levels at the wound site. [Antioxid Redox Signal] Abstract

Improved Efficacy and In Vivo Cellular Properties of Human Embryonic Stem Cell Derivative in a Preclinical Model of Bladder Pain Syndrome

Scientists showed greater therapeutic efficacy of human embryonic stem cell-derived multipotent stem cells (M-MSCs) than adult bone-marrow-derived counterparts for treating interstitial cystitis/bladder pain syndrome and also monitored long-term safety and in vivo properties of transplanted M-MSCs in living animals. [Sci Rep] Full Article

Novel Prostate Cancer Immunotherapy with a DNA-Encoded Anti-Prostate-Specific Membrane Antigen Monoclonal Antibody

The authors describe a novel strategy of antibody-based immunotherapy against prostate carcinoma that utilizes synthetic DNA plasmids that encode a therapeutic human monoclonal antibodies that target prostate-specific membrane antigen. [Cancer Immunol Immunother] Full Article

Minimally Manipulated Murine Regulatory T Cells Purified by Reversible Fab Multimers Are Potent Suppressors for Adoptive T Cell Therapy

Researchers showed that reversible Fab multimer-based regulatory T cell purification can prevent conventional antibody label-induced interferences in vitro and in vivo. [Eur J Immunol] Abstract

Gene Therapy with an AAV Vector Expressing Human IL-2 Alters Immune System Homeostasis in Humanized Mice

Humanized mice treated with an adeno associated virus (AAV) vector expressing human IL-2 showed a significant and sustained increase in the number of functional human FOXP3⁺CD4⁺regulatory T cells. [Hum Gene Ther] Abstract

Endothelial and Cardiac Progenitor Cells for Cardiovascular Repair: A Controversial Paradigm in Cell Therapy

The authors provide a summary of the different applications of endothelial progenitor cells and cardiac progenitor cells for cardiovascular cell therapy and underlie their advantages and limitations. [Pharmacol Ther] Abstract

Enhancing Human Regulatory T Cells In Vitro for Cell Therapy Applications

With a particular focus on regulatory T cells, scientists survey the diverse strategies that are being employed to enhance the efficacy of cell therapy via in vitro manipulation of cells, and highlight some emerging technologies that may propel this endeavor in the future. [Immunol Lett] Abstract

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

Physicians’ Education Resource^® to Host Three Satellite Symposia

Physicians’ Education Resource^® will conduct three CME-Accredited Satellite Symposia. The programs will focus on some of the most pressing issues in hematology. [Press release from Physicians’ Education Resource^® (Business Wire Inc.) discussing research to be presented at American Society of Hematology’s 59^th Annual Meeting & Exposition (ASH), Atlanta] Press Release

CRISPR Therapeutics Announces Collaboration with Massachusetts General Hospital to Research Use of CRISPR/Cas9 in T Cell Cancer Therapies

CRISPR Therapeutics and Massachusetts General Hospital Cancer Center have entered into a two-year research collaboration and license option agreement to develop novel T cell therapies for cancer. [CRISPR Therapeutics] Press Release

Sangamo and Pfizer Announce First Patient Receives Treatment in Phase I/II Clinical Trial Evaluating SB-525 Investigational Gene Therapy for Hemophilia A

Sangamo Therapeutics, Inc. and Pfizer Inc. announced that the first patient received treatment in the Phase I/II clinical trial evaluating SB-525, an investigational gene therapy for patients with hemophilia A, a rare genetic blood disorder. [Sangamo Therapeutics, Inc.] Press Release

Gilead Sciences to Acquire Kite Pharma for $11.9 Billion

Gilead Sciences, Inc. and Kite Pharma, Inc. announced that the companies have entered into a definitive agreement pursuant to which Gilead will acquire Kite for $180.00 per share in cash. [Gilead Sciences, Inc.] Press Release

Orchard Therapeutics Announces that OTL-101 Has Received a Designation as Promising Innovative Medicine by UK’s Regulatory Agency

Orchard Therapeutics Limited announced that the UK Medicines and Healthcare Products Regulatory Agency granted a Promising Innovative Medicine Designation to OTL-101, its lead programme for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. [Orchard Therapeutics Limited] Press Release

Immatics Initiates Personalized Adoptive Cellular Therapy in Patients with Relapsed and/or Refractory Solid Cancers Using Its Pioneering Target Warehouse

Immatics announced that it has initiated enrollment of patients into a Phase I trial of its first adoptive cellular therapy IMA101, using its proprietary ACTolog^® approach. [Immatics Biotechnologies GmbH] Press Release

CSL Behring to Acquire Biotech Company Calimmune and Its Proprietary Stem Cell Gene Therapy Platform

CSL Behring announced that it has agreed to acquire Calimmune, Inc., a biotechnology company focused on the development of ex vivo hematopoietic stem cell gene therapy with R&D facilities in Pasadena, California and Sydney, Australia for an upfront payment of $91 million. [CSL Behring (PR Newswire Association LLC.)] Press Release

MeiraGTx Provides Clinical Trial Updates for X-Linked Retinitis Pigmentosa and Achromatopsia Gene Therapy Programs

MeiraGTx announced the first patient in its clinical study for X-linked retinitis pigmentosa was treated at Moorfields Eye Hospital in London. [MeiraGTx Limited] Press Release

Apic Bio Launches to Advance First-in-Class Gene Therapy for Treatment of Alpha-1 Antitrypsin Deficiency (Alpha 1)

Apic Bio, Inc. launched with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease. [Apic Bio, Inc. (PR Newswire Association LLC.)] Press Release

FDA Warns US Stem Cell Clinic of Significant Deviations

The FDA posted a warning letter issued to US Stem Cell Clinic of Sunrise, Florida, and its Chief Scientific Officer Kristin Comella for marketing stem cell products without FDA approval and for significant deviations from current good manufacturing practice requirements, including some that could impact the sterility of their products, putting patients at risk. [U.S. Food and Drug Administration] Editorial

Statement from FDA Commissioner Scott Gottlieb, M.D. on the FDA’s New Policy Steps and Enforcement Efforts to Ensure Proper Oversight of Stem Cell Therapies and Regenerative Medicine

In the last few days alone, the FDA has taken steps in Florida and California to address a number of especially troubling products being marketed. But unfortunately, these are examples of a larger pool of actors who claim that their unproven and unsafe products will address a serious disease, but instead put patients at significant risk. [U.S. Food and Drug Administration] Editorial

Biomedical Ph.D. Program at Major Research University Drops GRE Requirement for Admission

The University of Michigan’s biomedical sciences graduate program announced that it will no longer require GRE scores for its Ph.D. admissions. Following a review of the available evidence and a public discussion involving the program’s faculty, staff, and trainees, the exam’s ability to predict student performance seems “weak at best” while it significantly disadvantages women, minorities, and students from low socioeconomic backgrounds, writes Scott Barolo, director of the Program in Biomedical Sciences, in the announcement. [Science Careers] Editorial

FDA

Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting (FR Doc. No:2017-18161) Notice

NIH

Government-Owned Inventions; Availability for Licensing (FR Doc. No:2017-18137) Notice

NEW Translational Stem Cell Research Conference
October 24-25, 2017
New York, New York, United States

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

NEW Technologist – Cell Therapy (Yale New Haven Health)

NEW Researcher – Skeletal Tissue Engineering (KU Leuven)

Postdoctoral Fellow – Cell Biology, Tumor Immunology and Targeted Therapy (University of Minnesota)

Postdoctoral Associate – Enhancer RNA Therapy (Sylvester Comprehensive Cancer Center)

Postdoctoral Fellow – Cell and Gene Therapy Studies Targeting Heart Failure (Icahn School of Medicine at Mount Sinai)

Research Group Leaders (Centro de Investigación Príncipe Felipe)

Postdoctoral Research Associate – Molecular Mechanisms of Stem Cells in the Heart (University of Louisville)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

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

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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