Cell Therapy News Volume 18.24 | Jul 17 2017

TOP STORY

COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa

The authors have exploited an ex vivo CRISPR/Cas9-mediated homology-directed repair approach for the correction of a frequent inherited mutation in exon 80 of COL7A1, which impairs type VII collagen expression, causing the severe blistering skin disease recessive dystrophic epidermolysis bullosa. [Mol Ther] Abstract | Full Article

(Ranked by impact factor of the journal)

Long-Duration Complete Remissions of Diffuse Large B-Cell Lymphoma after Anti-CD19 Chimeric Antigen Receptor Therapy

Scientists administered anti-CD19 CAR T cells preceded by cyclophosphamide and fludarabine conditioning chemotherapy to patients with relapsed diffuse large B-cell lymphoma. Five of the seven evaluable patients obtained complete remissions. [Mol Ther] Abstract

Oncolytic Measles Virus Enhances Antitumor Responses of Adoptive CD8⁺NKG2D⁺ Cells in Hepatocellular Carcinoma Treatment

Researchers investigated the potency of the measles virus vaccine strain Edmonston in improving adoptive CD8⁺NKG2D⁺ cells for hepatocellular carcinoma treatment. [Sci Rep] Full Article

Prospectively Isolated Mesenchymal Stem/Stromal Cells Are Enriched in the CD73⁺ Population and Exhibit Efficacy after Transplantation

On the basis of CD29 and CD54 expression, scientists prospectively isolated a population with a high colony-forming ability and multi-lineage potential from the rat bone marrow, and they demonstrated that most of these cells expressed CD73. Successful engraftment of rat mesenchymal stem/stromal cells was achieved by using a fluorescence-conjugated anti-CD73 antibody. [Sci Rep] Full Article

Allogeneic Hematopoietic Stem Cell Transplantation with Myeloablative Conditioning Is Associated with Favorable Outcomes in Mixed Phenotype Acute Leukemia

The authors present their experience of genomic profiling, pre-transplant therapy and transplant outcomes for 36 well-characterized pediatric and adult patients with mixed phenotype acute leukemia, defined according to the 2016 WHO leukemia update. [Biol Blood Marrow Transplant] Abstract

Stromal Vascular Fraction Loaded Silk Fibroin Mats Effectively Support the Survival of Diabetic Mice after Pancreatic Islet Transplantation

Investigators assessed whether stromal vascular fraction-soaked silk fibroin nonwoven mats can preserve the functionality of encapsulated pancreatic endocrine cells after transplantation in the subcutaneous tissue of diabetic mice. [Macromol Biosci] Abstract

Pericytes Extend Survival of ALS SOD1 Mice and Induce the Expression of Antioxidant Enzymes in the Murine Model and in iPSCs Derived Neuronal Cells from an ALS Patient

Researchers evaluated the therapeutic effect of human pericytes in vivo in SOD1 mice and in vitro in motor neurons and other neuronal cells derived from one amyotrophic lateral sclerosis (ALS) patient. [Stem Cell Rev] Abstract | Graphical Abstract

Mesenchymal Stem Cell Transplantation Carried in SVVYGLR Modified Self-Assembling Peptide Promoted Cardiac Repair and Angiogenesis after Myocardial Infarction

Scientists designed a new self-assembling peptide through adding angiogenic polypeptide SVVYGLR to the carboxyl terminal of RADA16, and evaluated the therapeutic potential of mesenchymal stem cell transplantation carried in this designer self-assembling peptide on myocardial infarction. [Biochem Biophys Res Commun] Abstract

Autologous Bone Marrow-Derived Mononuclear Cells Transplantation in Type 2 Diabetes Mellitus: Effect on β-Cell Function and Insulin Sensitivity

Investigators examined the efficacy and safety of autologous bone marrow-derived mononuclear cells transplantation in type 2 diabetes mellitus, and explored the mechanistic insights into stem cells action through metabolic studies. [Diabetol Metab Syndr] Full Article

Mesenchymal Stem Cells and Their Immunosuppressive Role in Transplantation Tolerance

The authors discuss some of the contemporary evidence for use of mesenchymal stem cells in the field of immune tolerance, with a special emphasis on transplantation. [Ann N Y Acad Sci] Abstract

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Novartis CAR-T Cell Therapy CTL019 Unanimously (10-0) Recommended for Approval by FDA Advisory Committee to Treat Pediatric, Young Adult r/r B-Cell ALL

Novartis announced that the FDA Oncologic Drugs Advisory Committee unanimously recommended approval of CTL019, an investigational CAR-T therapy, for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). [Novartis AG] Press Release | Editorial

Asterias Biotherapeutics Completes Enrollment and Dosing of SCiStar Study’s AIS-B 10 Million Cell Cohort

Asterias Biotherapeutics, Inc. announced completion of enrollment and dosing of the AIS-B 10 million cell cohort in the company’s ongoing SCiStar Phase I/IIa clinical study of AST-OPC1 in complete cervical spinal cord injury. [Asterias Biotherapeutics, Inc.] Press Release

BioMarin’s Investigational Gene Therapy for Hemophilia A at 6e13 vg/kg Dose Maintains Average Factor VIII Levels within Normal Range for over One Year

BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase I/II study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. [BioMarin Pharmaceutical Inc.] Press Release

Spark Therapeutics’ Biologics License Application for Investigational Voretigene Neparvovec Accepted for Filing by FDA

Spark Therapeutics announced that the FDA has accepted for filing the Biologics License Application and granted Priority Review for voretigene neparvovec, an investigational, potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease. [Spark Therapeutics, Inc.] Press Release

Sangamo Receives Fast Track Designation from the FDA for SB-318 and SB-913 In Vivo Genome Editing Product Candidates for the Treatment of MPS I and MPS II

Sangamo Therapeutics, Inc. announced that the FDA has granted Fast Track designation to SB-318 and SB-913, the company’s clinical stage in vivo genome editing product candidates for the treatment of Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively. [Sangamo Therapeutics, Inc.] Press Release

Korea Approves the World’s First Cell and Gene Therapy for Knee Osteoarthritis

TissueGene, Inc. announced that Kolon Life Science has received marketing approval for Invossa-K Inj., the world’s first cell and gene therapy for degenerative arthritis from the Korea Ministry of Food and Drug Safety. [TissueGene, Inc.] Press Release

Two Female Scientists Sue Salk Institute, Alleging Discrimination at ‘Old Boys Club’

Two senior female scientists are suing their employer, the prestigious Salk Institute for Biological Studies, alleging pervasive, long-standing gender discrimination. In the pair of lawsuits, filed in California Superior Court in San Diego, plaintiffs Vicki Lundblad and Katherine Jones seek unspecified compensation for an array of harms. [ScienceInsider] Editorial

To Woo Public, Europe Opens Up on Animal Experiments, but U.S. Less Transparent

A London-based group that supports the use of animals in biomedical science began inviting the public to take an unusual digital tour of laboratories at four U.K. research institutions. At LabAnimalTour.org, users can watch a monkey with a bolt in its skull forage in its cage at a University of Oxford neuroscience lab and a technician check on some of the 8000 mice housed in one room at the Medical Research Council’s Harwell Institute. [ScienceInsider] Editorial

Why a Flat 2018 Budget Could Tie NSF’s Hands

What would a flat budget mean for the National Science Foundation (NSF)? For many agencies, it means maintaining the status quo. And NSF may have trouble doing anything other than that thanks to some pointed instructions from a key legislator. [ScienceInsider] Editorial

FDA

Product-Specific Guidances; Draft and Revised Draft Guidances for Industry; Availability (FR Doc. No:2017-14781) Notice

NIH

Prospective Grant of Exclusive Patent License: Development, Commercial Application and Use of Fulvestrant in Combination Therapy for the Treatment of Cancers (FR Doc. No:2017-14860) Notice

Prospective Grant of Exclusive Patent License: Composition and Methods for Delivering Inhibitory Oligonucleotides for the Treatment of Pancreatic Cancer (FR Doc. No:2017-14370) Notice

NEW Advances in Modeling Cancer in Mice: Technology, Biology, and Beyond
September 24-27, 2017
Orlando, Florida, United States

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NEW Postdoctoral Research Associate – Gene Therapy (King’s College London)

Senior Research Associate – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Agency Research Center)

Research Associate – Cell Engineering (Caribou Biosciences, Inc.)

PhD Scholarship – Virology/Gene Therapy (University of New South Wales)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Scientist – Analytical Development (KBI Biopharma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

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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