Cell Therapy News Volume 19.16 | May 7 2018

TOP STORY

Transfer of Gene Corrected T Cells Corrects Humoral and Cytotoxic Defects in X-Linked Lymphoproliferative Disease (XLP1)

CD3⁺ lymphocytes from sap-deficient mice were transduced with a gammaretroviral vector encoding human SAP cDNA before transfer into sub-lethally irradiated sap-deficient recipients. [J Allergy Clin Immunol] Abstract

(Ranked by impact factor of the journal)

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Researchers demonstrated the use of cell therapy as a potential long-term cure for hemophilia B in our factor IX (FIX)-deficient mouse model. They showed that transplanted, cryopreserved, cadaveric human hepatocytes remain functional for more than a year and secrete FIX at therapeutic levels. [Cell Rep] Full Article | Press Release | Graphical Abstract

Grafts of Olfactory Stem Cells Restore Breathing and Motor Functions after Rat Spinal Cord Injury

Using an acute rat model of high cervical contusion that provokes a persistent hemidiaphragmatic and foreleg paralysis, the authors evaluated the therapeutic effect of a delayed syngeneic transplantation of olfactory ecto-mesenchymal stem cells within the injured spinal cord. [J Neurotrauma] Abstract

Chondrogenic Differentiation Processes in Human Bone Marrow Aspirates Seeded in Three-Dimensional Woven Poly(ε-Caprolactone) Scaffolds Enhanced by rAAV-Mediated SOX9 Gene Transfer

Scientists evaluated the effect of genetically modifying concentrated human mesenchymal stem cells from bone marrow to induce chondrogenesis by recombinant adeno-associated viral (rAAV) vector gene transfer of the sex-determining region Y-type high-mobility group box 9 (SOX9) factor upon seeding in three-dimensional woven poly(ε-caprolactone) scaffolds that provide mechanical properties mimicking those of native articular cartilage. [Hum Gene Ther] Abstract

Administration of Cripto in GRP78 Overexpressed Human MSCs Enhances Stem Cell Viability and Angiogenesis during Human MSC Transplantation Therapy

Investigators explored the effectiveness of concurrent GRP78 overexpression combined with Cripto on hMSC proliferation and migration both in vitro and in vivo. They explored whether the treatment enhances effectiveness of hMSC transplantation in ischemic tissue. [Cell Prolif] Abstract

Mesenchymal Stem Cell Sheets Exert Anti-Stenotic Effects in a Rat Arterial Injury Model

A rat model of femoral artery injury was used to compare the anti-stenotic effects of transplanted cell sheets and transplanted cell suspensions. Rat adipose-derived stem cells were used as the source of mesenchymal stem cells. [Tissue Eng Part A] Abstract

Regulatable Lentiviral Hematopoietic Stem Cell Gene Therapy in a Mouse Model of Parkinson’s Disease

Researchers developed a hematopoietic stem cell transplantation-based macrophage-mediated glial cell line-derived neurotrophic factor (GDNF) therapy platform. They introduced a regulatable lentiviral vector to allow the expression of human GDNF to be adjusted or stopped by oral administration of doxycycline. [Stem Cells Dev] Abstract

Autologous Stem Cell Transplantation for Refractory Opsoclonus Myoclonus Ataxia Syndrome

Investigators present two cases who were refractory to conventional therapy and underwent autologous stem cell transplantation (ASCT). One patient had complete resolution of symptoms following ASCT and the other patient had minimal change in symptoms with this therapy. [Pediatr Blood Cancer] Abstract

Mesenchymal Stem Cells Form 3D Clusters following Intraventricular Transplantation

Scientists investigated the in vivo behavior of canine, human, and murine mesenchymal stem cells following intra-cerebroventricular transplantation. [J Mol Neurosci] Abstract

Promoter-Operating Targeted Expression of Gene Therapy in Cancer: Current Stage and Prospect

The authors clarify the current state and some problems in the development of promoter-operating targeted expression of interesting genes and highlight its potential in cancer gene therapy. [Mol Ther Nucleic Acids] Abstract

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

Rocket Pharmaceuticals Announces Presentations

Rocket Pharmaceuticals, Inc. announced eight oral and poster presentations. [Press release from Rocket Pharmaceuticals, Inc. discussing research to be presented at the 2018 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Chicago] Press Release

Editas Medicine to Present New Data Demonstrating Progress towards Creating Genome Editing Medicines

Editas Medicine, Inc. announced that ten scientific abstracts, including three from research collaborations, have been accepted for presentation. [Press release from Editas Medicine, Inc. discussing research to be presented at the 2018 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Chicago] Press Release

AstraZeneca, SWIBCo Launch Stem Cell Cardiovascular Therapy Collaboration

AstraZeneca will develop novel cardiovascular and catheter-based therapies using Procella Therapeutics’ stem cell technology and Smartwise’s catheter injection method. [AstraZeneca (Genetic Engineering & Biotechnology News)] Press Release

Cancer Center at BIDMC Launches Immunotherapy Institute

The Cancer Center at Beth Israel Deaconess Medical Center (BIDMC) announced the launch of a new clinical and research institute that will build on BIDMC’s long history of leadership in immunotherapy and cell therapeutics. The Immunotherapy Institute will harness the potential of immunotherapy as it revolutionizes the way cancer and ultimately other diseases are treated and diagnosed. [Beth Israel Deaconess Medical Center] Press Release

4DMT and ROCHE Expand Ophthalmology Partnership to Develop and Commercialize Multiple AAV Gene Therapy Products

4D Molecular Therapeutics announced the expansion of its 2015 research agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. into a broad long-term partnership to develop and commercialize multiple ophthalmology products. [4D Molecular Therapeutics] Press Release

Ferring Signs Global Agreement to Commercialize Novel Gene Therapy for Bladder Cancer Patients

Ferring Pharmaceuticals announced the signing of an agreement giving the company the option to secure global commercialization rights to nadofaragene firadenovec/Syn3, a novel gene therapy being developed by FKD Therapies Oy as a treatment for patients with high-grade non-muscle invasive bladder cancer, who are unresponsive to Bacillus Calmette-Guérin therapy. [Ferring B.V.] Press Release

Sarepta Therapeutics Announces Partnership with Myonexus Therapeutics for the Advancement of Multiple Gene Therapy Programs Aimed at Treating Distinct Forms of Limb-Girdle Muscular Dystrophies

Sarepta Therapeutics, Inc. announced that it has entered into an exclusive partnership with Myonexus Therapeutics, Inc. [Sarepta Therapeutics, Inc.] Press Release

Hackensack Meridian Health Joins NJII’s Cell and Gene Therapy Development Center

The New Jersey Innovation Institute (NJII) has announced that Hackensack Meridian Health has joined its Cell and Gene Therapy Development Center to access the center’s process development and clinical manufacturing capabilities. [Hackensack Meridian Health] Press Release

Kymriah^® (Tisagenlecleucel), First-in-Class CAR-T Therapy from Novartis, Receives Second FDA Approval to Treat Appropriate r/r Patients with Large B-Cell Lymphoma

Novartis announced the FDA has approved Kymriah^® suspension for intravenous infusion for its second indication – the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma and DLBCL arising from follicular lymphoma. [Novartis Pharmaceuticals Corporation] Press Release

FDA Backs Gene Therapy for Fatal Neurodegenerative Childhood Disease

Orchard Therapeutics received a Rare Pediatric Disease Designation form the FDA for its gene therapy OTL-200. The treatment is for patients with metachromatic leukodystrophy, a fatal genetic disease that causes sulfur to accumulate in the body, harming the nervous system and metabolism. [Orchard Therapeutics (Labiotech UG)] Press Release

REGENXBIO Receives FDA Fast Track Designation for RGX-121 Gene Therapy for the Treatment of Mucopolysaccharidosis Type II

REGENXBIO Inc. announced that the FDA has granted Fast Track designation for RGX-121. [REGENXBIO Inc.] Press Release

Immusoft Granted Orphan Drug Designation for Treatment of Rare Childhood Disease

Immusoft Corporation announced that the FDA has granted Orphan Drug Designation to treat the first indication in the company’s pipeline, mucopolysaccharidosis type I. [Immusoft Corporation (Business Wire, Inc.)] Press Release

Alnylam Achieves Alignment with FDA on Accelerated Development Path for Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1 (PH1)

Alnylam Pharmaceuticals, Inc. announced that the company has reached alignment with the FDA on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of PH1. [Alnylam Pharmaceuticals, Inc.] Press Release

Hemostemix Announces First Patient Treated in Phase II Clinical Trial

Hemostemix Inc. announced that it has treated its first patient under its continuing Phase II Clinical Trial for critical limb ischemia. [Hemostemix Inc.] Press Release

Celyad Successfully Administers CYAD-01 in First Patients in SHRINK and LINK Trials

Celyad announced the successful injection of the first patients in the SHRINK trial and the LINK trial, both targeting metastatic colorectal patients. [Celyad] Press Release

Mackall Awarded $11.9 Million for Anti-Leukemia Clinical Trial

Crystal Mackall, professor of pediatrics and of medicine at the School of Medicine, was awarded $11.9 million by the governing board of the California Institute for Regenerative Medicine to fund a clinical trial of immune cells genetically modified to recognize two proteins on the surface of leukemia and lymphoma cells. [Stanford University] Press Release

Basic Scientists Still Feel Pinch of New NIH Clinical Trial Policy

Basic researchers who study the brain and human behavior thought lawmakers had come to their rescue in March by blocking the National Institutes of Health (NIH) from redefining their studies as clinical trials. But NIH officials are still pushing ahead with new requirements that scientists say make no sense and will cripple their research. [ScienceInsider] Editorial

Petition Asks National Academy of Sciences to Boot Sexual Harassers

An online petition calling on the National Academy of Sciences to revoke memberships bestowed on people sanctioned for sexual harassment has garnered more than 250 signatures since it was posted on May 1. [The Scientist] Editorial

Nature Announces New Editor-in-Chief

Nature has named Magdalena Skipper as its new editor-in-chief. She is the first woman to hold the post. Skipper, who is currently editor-in-chief of the open-access journal Nature Communications, will become the eighth editor of Nature. [Nature News] Editorial

Big UK Science Funder Says Report Harassment or Risk Losing Funding

One of the world’s largest research-funding charities is cracking down on harassment and bullying. Scientists who have been sanctioned by their institutions could lose out on funding from the Wellcome Trust, under rules announced. [Nature News] Editorial

NIH

Prospective Grant of Exclusive Patent License: Antibodies against TL1A, a TNF-Family Cytokine, for the Treatment and Diagnosis of Crohn’s Disease, Ulcerative Colitis, Asthma, Psoriasis and Biliary Cirrhosis (FR Doc. No:2018-09654) Notice

NEW Federation of American Societies for Experimental Biology (FASEB): Skeletal Muscle Satellite Cells and Regeneration
July 8-13, 2018
Steamboat Springs, Colorado, United States

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

NEW Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

NEW Postdoctoral Position – Technology-Driven Research in Biomedicine (CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences)

NEW Group Manager – AAV Vector Expression Systems (Genethon)

Postdoctoral Fellow – Gene Therapy in Cardiovascular Disease (Emory University)

Research Associate – Gene and Cell Therapy (University College London)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Scientist/Senior Scientist – Bioinformatics and Data Sciences (Obsidian Therapeutics)

Scientist – Discovery Biology (Prevail Therapeutics)

Postdoctoral Fellow – Precision Therapy of Aggressive Lymphoma (AstraZeneca)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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