Volume 17.17 | May 2

Issue 17.17 May 2, 2016
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Gene Therapy Shows Long-Term Benefit for Treating Rare Blindness
Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new research shows. [Press release from the University of Oxford discussing online prepublication in New England Journal of Medicine] Press Release | Full Article
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PUBLICATIONS (Ranked by impact factor of the journal)
Conversion of Human Fibroblasts into Functional Cardiomyocytes by Small Molecules
Investigators showed that cardiomyocyte-like cells can be generated by treating human fibroblasts with a combination of nine compounds (9C). The chemically induced cardiomyocyte-like cells (ciCMs) uniformly contracted and resembled human cardiomyocytes in their transcriptome, epigenetic, and electrophysiological properties. When transplanted into infarcted mouse hearts, 9C-treated fibroblasts were efficiently converted to ciCMs. [Science] Full Article | Press Release

Chemical Control of Grafted Human PSC-Derived Neurons in a Mouse Model of Parkinson’s Disease
Scientists showed tunable rescue of motor function in a mouse model of Parkinson’s disease, following transplantation of human midbrain dopaminergic neurons differentiated from human pluripotent stem cells engineered to express designer receptors exclusively activated by designer drug. [Cell Stem Cell]
Abstract | Press Release | Graphical Abstract

Intratracheal Gene Delivery of SERCA2a Ameliorates Chronic Post-Capillary Pulmonary Hypertension
Scientists assessed the feasibility of sustained pulmonary vascular sarcoplasmic reticulum Ca2+-ATPase 2a (SERCA2a) gene expression using aerosolized delivery of adeno-associated virus type 1 in a large animal model of chronic pulmonary hypertension and evaluated the efficacy of gene transfer regarding progression of pulmonary vascular and right ventricular remodeling. [J Am Coll Cardiol] Abstract | Press Release

CD19 CAR–T Cells of Defined CD4+:CD8+ Composition in Adult B Cell ALL Patients
Researchers conducted a clinical trial to evaluate CD19 chimeric antigen receptor (CAR)–T cells that were manufactured from defined CD4+ and CD8+ T cell subsets and administered in a defined CD4+:CD8+ composition to adults with B cell acute lymphoblastic leukemia (ALL) after lymphodepletion chemotherapy. [J Clin Invest] Full Article | Press Release

miR-22 Has a Potent Anti-Tumor Role with Therapeutic Potential in Acute Myeloid Leukaemia
Scientists showed that miR-22 is an essential anti-tumor gatekeeper in de novo acute myeloid leukemia where it is significantly downregulated. Forced expression of miR-22 significantly suppressed leukaemic cell viability and growth in vitro, and substantially inhibited leukemia development and maintenance in vivo. [Nat Commun] Full Article | Press Release

Superior Therapeutic Index in Lymphoma Therapy: CD30+ CD34+ Hematopoietic Stem Cells Resist a Chimeric Antigen Receptor (CAR) T Cell Attack
Recent clinical trials with CAR redirected T cells targeting CD19 revealed particular efficacy in the treatment of leukemia/lymphoma, however, were accompanied by a lasting depletion of healthy B cells. The authors explored CD30 as an alternative target which is validated in lymphoma therapy and expressed by a broad variety of Hodgkin’s and non-Hodgkin’s lymphomas. [Mol Ther] Abstract

Bone Marrow-Derived Mesenchymal Stem Cells Improve Diabetes-Induced Cognitive Impairment by Exosome Transfer into Damaged Neurons and Astrocytes
Scientists showed that rat bone marrow mesenchymal stem cells can improve the cognitive impairments of STZ-diabetic mice by repairing damaged neurons and astrocytes. [Sci Rep] Full Article

AAV-mediated Transduction and Targeting of Retinal Bipolar Cells with Improved mGluR6 Promoters in Rodents and Primates
Researchers showed that the combination of the endogenous mGluR6 promoter with additional enhancers in the introns of the mGluR6 gene markedly enhanced adeno-associated virus (AAV) transduction efficiency as well as made the targeting more selective for rod bipolar cells in mice. [Gene Ther] Abstract

Ex Vivo Expansion of Circulating CD34+ Cells Enhances the Regenerative Effect on Rat Liver Cirrhosis
Researchers aimed to investigate the efficacy of human ex vivo-expanded CD34+ cells for treatment of cirrhotic rat liver. [Mol Ther Methods Clin Dev] Full Article

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Cardiac Gene Therapy: Are We There Yet?
The reviewers summarize the gene delivery carriers, modes of delivery, recent preclinical/clinical studies and potential therapeutic targets. They briefly discuss the existing limitations of gene therapy, technical challenges surrounding gene carriers and delivery systems, and some approaches to overcome these limitations for bringing cardiovascular disease gene therapy one step closer to reality. [Gene Ther] Abstract

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

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Phase II Cervical Cancer Study with Advaxis’ Axalimogene Filolisbac Selected for Poster Discussion Session
The poster, “ADXS11-001 immunotherapy in squamous or non-squamous persistent/recurrent metastatic cervical cancer: Results from stage I of the Phase II GOG/NRG0265 study,” was selected as one of 12 abstracts to be featured. [Press release from Advaxis, Inc. discussing research to be presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Heat Biologics and OncoSec Present Data
Heat Biologics, Inc. announced that preclinical data from its collaboration with OncoSec Medical Incorporated focused on evaluating the combination of immunotherapy platforms were presented. [Press release from Heat Biologics, Inc. discussing research presented at the 2016 American Association for Cancer Research (AACR) Annual Meeting, New Orleans] Press Release

Vycellix’s CEO to Present Novel Approaches to Adoptive Cancer Immunotherapy
Vycellix, Inc. announced that its CEO, Evren Alici, M.D., Ph.D., will present. The company expects to initially advance its technology for the treatment of hematological malignancies and sarcomas. [Press release from Vycellix, Inc. discussing research presented at the Innate Killer Summit 2016, San Diego] Press Release

Asterias Biotherapeutics Announces Oral Presentation
Asterias Biotherapeutics, Inc. announced that clinical data from its AST-VAC1 (antigen-presenting autologous dendritic cells) immunotherapy clinical program will be presented during an oral session. [Press release from Vycellix, Inc. discussing research presented at the American Society of Gene and Cell Therapy (ASGCT) 19th Annual Meeting, Washington, D.C.] Press Release

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Researchers Push for Personalized Tumor Vaccines
It is precision medicine taken to the extreme: cancer-fighting vaccines that are custom designed for each patient according to the mutations in their individual tumors. With early clinical trials showing promise, that extreme could one day become commonplace — but only if drug developers can scale up and speed up the production of their tailored medicines. [Nature News] Editorial

U.S. And U.K. Universities Announce Binational Postdoc Program
Ph.D. scientists looking for a different kind of PostDoc may wish to consider applying for an unusual new fellowship announced 14 April by the University of Birmingham in the United Kingdom and the University of Illinois, Urbana-Champaign (UIUC), in the United States. The appointments last 3 years, with the first and third spent at Birmingham and the second at UIUC. Fellows will have a mentor at each institution. [Science] Editorial

Global Stem Cells Group and University of Santiago Biotech Lab Endorse Asia-Pacific Symposium
Global Stem Cells Group and the University of Santiago Biotechnology Lab have announced a mutual endorsement of an Asia-Pacific Symposium as other research and development initiatives for potential stem cell protocol management for 2016 – 2020. [Global Stem Cells Group] Editorial

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Homology Medicines Inc. Launches as a Newly-Formed Genetic Medicines Company Founded Upon a Unique City of Hope Gene Editing and Gene Transfer Platform Technology
City of Hope announced that it has licensed its pioneering AAV-based gene editing technology exclusively to Homology Medicines Inc. A City of Hope lab led by Saswati Chatterjee, Ph.D., developed the single platform technology that enables both gene editing and gene therapy with a built-in in vivo delivery system. [City of Hope (Business Wire)] Press Release

Regenerating Blood Vessels Gets $2.7 Million Grant
Biomedical engineers at The University of Texas at Austin have received a three-year, $2.7 million grant from the U.S. Department of Defense to advance a promising treatment that could help millions of people with diabetes affected by peripheral ischemia, a condition that restricts blood flow to muscles in the lower limbs. [The University of Texas at Austin] Press Release

Queen’s Researchers in €2.25m International Project to Tackle Diabetes-Related Blindness
The new €2.25m US-Ireland R&D partnership is bringing together a unique team of scientists and clinicians to develop a gene therapy approach with the aim of potentially reversing diabetes-linked blood vessel damage to the retina – the light-sensitive tissue at the back of the eye. Ultimately, the team hopes to prevent disease progression and restore visual function. [Queen’s University Belfast] Press Release

TRACT Therapeutics, Inc. Received FDA Approval for a Phase II Trial Using a Patient’s Immune Cells to Prevent Organ Rejection
TRACT Therapeutics, Inc. announced that it received Food and Drug Administration (FDA) approval to initiate a Phase II clinical trial to investigate the efficacy and safety of its novel therapy for kidney transplant recipients. [TRACT Therapeutics, Inc. (Business Wire)] Press Release

Regenerating Brain Tissue Brings Hope for a New Treatment against Alzheimer’s and Parkinson’s
Mexican researchers developed a nanogel that enables the growth of new neurons. Working with animal models, Mexican researchers were able to cross the electroencephalic barrier, introduce a nanogel and achieve the growth of neurons within the gel, which shows that it is possible to promote regeneration of brain tissue. [Investigación y Desarrollo (AlphaGalileo)] Press Release

First Gene Therapy Successful Against Human Aging
The 44 year-old CEO of BioViva USA Inc. Elizabeth Parrish received two of her own company’s experimental gene therapies: one to protect against loss of muscle mass with age, another to battle stem cell depletion responsible for diverse age-related diseases and infirmities. [BioViva USA Inc.] Press Release

Dr. Patrick Soon-Shiong Honored at the Vatican with the 2016 Pontifical Key Visionary Award
Dr. Patrick Soon-Shiong, who grew up in Apartheid South Africa and has dedicated his life to pioneering novel cellular therapies for diabetes and cancer, including the development of the revolutionary cancer drug Abraxane, was honored at the Vatican with the 2016 Pontifical Key Visionary Award. [Business Wire] Press Release

University of Wisconsin and Harvard Scientists Receive Awards from ASNTR
ASNTR awarded The 2016 Bernard Sanberg Memorial Award for Brain Repair to Marina E. Emborg, MD, PhD, an associate professor of medical physics at the University of Wisconsin-Madison. ASNTR also presented The Molly and Bernard Sanberg Memorial Award to Eng H. Lo, PhD, professor of neurology and radiology at Harvard University and Massachusetts General Hospital. [University of South Florida (EurkeAlet!)] Press Release
Prospective Grant of Exclusive License: AAV-Mediated Aquaporin Gene Transfer To Treat Sjögren’s Syndrome (FR Doc. No: 2016-09978)

Prospective Grant of Exclusive License: The Development of Anti-CD70 Chimeric Antigen Receptors (CARs) for the Treatment of Chronic Myelogenous Leukemia (FR Doc. No: 2016-09324)
Center for Biologics Evaluation and Research eSubmitter Program for Electronic Submission of Postmarketing Adverse Event Reports for Human Vaccine Products
Food and Drug Administration (United States)

Assay Development and Validation for Immunogenicity Testing of Therapeutic Protein Products; Revised Draft Guidance for Industry; Availability (FR Doc. No: 2009-D-0539)
NEW iPSC Maintenance and Neuronal Culture Derivation Training Course
June 26-26, 2016
San Francisco, United States

NEW Royan International Twin Congress: Reproductive Biomedicine & Stem Cell
August 31-September 2, 2016
Tehran, Iran

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW PhD Studentship – Various Projects (Vienna Biocenter)

NEW Postdoctoral Fellow – Stem Cell Models of Parkinson’s (University of Oxford)

NEW Group Leader – Development and Stem Cells (Monash University)

Scientist – Bioengineering (STEMCELL Technologies Inc.)

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

Postdoctoral Fellow – Immunology (The Pirbright Institute)

Full Professorship – Radiopharmaceutical Development (German Cancer Research Center)

Director – Stem Cells (PharmaScouts)

PhD Studentship – Stem Cell Biology (Masaryk University)

Assistant Professor – Cell Factory Design (Chalmers University of Technology)

Postdoctoral Fellow – Non-Viral Gene Delivery (Nanyang Technological University)

Project Lead – Engineering (Cytonome)

Faculty Position – Tumor Immunology (John Wayne Cancer Institute at Providence Saint John’s Health Center)

Postdoctoral Fellow – Non-Coding RNAs in Malignant Progression (The University of Texas MD Anderson Cancer Center)

Postdoctoral Associate – Stem Cell Biology (University of Illinois Chicago College of Medicine)

Postdoctoral Associate – Genetic Medicine (Cornell University)

Postdoctoral Fellow – Retinal Disease Genomics and Gene Therapy (Ocular Genomics Institute, Mass Eye and Ear and Harvard Medical School)

Postdoctoral Fellow – T Cell Reconstitution (Fred Hutchinson Cancer Research Center)

Associate Scientist – Hematopoietic Stem Cell Biology (Editas Medicine)

Research Associate – Hematopoietic Stem Cell Biology (Editas Medicine)

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

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

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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