Cell Therapy News Volume 20.26 | Jul 29 2019

TOP STORY

A Mutation-Independent Approach for Muscular Dystrophy via Upregulation of a Modifier Gene

Scientists report a mutation-independent strategy to upregulate the expression of a disease-modifying gene associated with congenital muscular dystrophy type 1A using the CRISPR activation system in mice. [Nature] Abstract | Press Release

(Ranked by impact factor of the journal)

AAV-Delivered eCD4-Ig Protects Rhesus Macaques from High-Dose SIVmac239 Challenges

Researchers investigated the ability of eCD4-Ig, an antibody-like entry inhibitor that emulates the HIV-1 and simian immunodeficiency virus (SIV) receptor and coreceptor, to prevent SIVmac239 infection. [Sci Transl Med] Abstract | Press Release

Phase III DREAM-HF Trial of Mesenchymal Precursor Cells in Chronic Heart Failure

The objective of DREAM-HF was to confirm earlier Phase II results and evaluate whether mesenchymal precursor cells will reduce the rate of nonfatal recurrent heart failure-related major adverse cardiac events while delaying or preventing progression of HF to terminal cardiac events. [Circ Res] Abstract

N-Cadherin Overexpression Enhances the Reparative Potency of Human iPSC-Derived Cardiac Myocytes in Infarcted Mouse Hearts

N-cadherin (CDH2) overexpression enhanced not only the survival/engraftment of cultured CDH2-human induced pluripotent stem cell-derived cardiac myocytes (hiPSC-CMs), but also the functional integration of these cells, consequently, the augmentation of the reparative properties of implanted CDH2-hiPSC-CMs in the failing hearts. [Cardiovasc Res] Abstract

Transcutaneous Ultrasound-Mediated Nonviral Gene Delivery to the Liver in a Porcine Model

Investigators developed a transhepatic venous nonviral gene delivery protocol in combination with transcutaneous, therapeutic ultrasound to facilitate significant gene transfer in pig livers. [Mol Ther Methods Clin Dev] Abstract | Full Article

A Biomimetic Self-Assembling Peptide Promotes Bone Regeneration In Vivo: A Rat Cranial Defect Study

Researchers hypothesised that addition of mesenchymal stromal cells would enhance the in vivo effects of P₁₁-4 in promoting skeletal tissue repair. [Bone] Abstract

Natural Killer Cells Impede the Engraftment of Cardiomyocytes Derived from Induced Pluripotent Stem Cells in Syngeneic Mouse Model

The authors demonstrated that natural killer cells (NKCs) infiltrated in induced pluripotent stem cell-derived cardiomyocyte (iPSC-CM) transplants even in a syngeneic mouse model. The depletion of NKCs using an anti-NKC antibody rescued transplanted iPSC-CMs, suggesting that iPSC-CMs activated NKC-mediated innate immunity. [Sci Rep] Full Article

In Vitro and In Vivo Effects of Insulin-Producing Cells Generated by Xeno-Antigen Free 3D Culture with RCP Piece

The hyperglycemic state of streptozotocin-induced diabetic nude mice converted to normoglycemic state around 14 days after transplantation of 96 insulin-producing cells under kidney capsule or intra-mesentery. [Sci Rep] Full Article

Cancer Therapy with MHC-Deficient and Interferon β-Producing Myeloid Cells Derived from Allogeneic Embryonic Stem Cells

The authors used the embryonic stem (ES) cell-derived myeloid cell line producing IFN ES-ML/IFN to treat allogeneic BALB/c mice transplanted with Colon26 cancer cells. Treatment with β-ML but not with γ-ML repressed growth of colon cancer in the peritoneal cavity and liver. Transferred ES-ML/IFN infiltrated into cancer tissues and enhanced infiltration of T cells into cancer tissues. [Cancer Sci] Abstract

Comparation of CART19 and Autologous Stem-Cell Transplantation for Refractory/Relapsed Non-Hodgkin’s Lymphoma

The chimeric antigen receptor T (CAR-T) group exhibited better rates of CR and one-year OS compared with the autologous stem-cell transplantation group. [JCI Insight] Abstract

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Mechanisms of Immunotherapy Resistance: Lessons from Glioblastoma

Investigators review the mechanisms of immunotherapy resistance in glioblastoma (GBM) and discuss how insights into GBM-immune system interactions might inform the next generation of immunotherapeutics for GBM and other resistant pathologies. [Nat Immunol] Abstract

Advances and Challenges for Hemophilia Gene Therapy

Adeno-associated viral based gene therapy is one of a number of novel approaches for treatment of hemophilia with other gene therapy and non-replacement therapies progressing through clinical trials. [Hum Mol Genet] Abstract

Immunotherapy for Acute Myeloid Leukemia: From Allogeneic Stem Cell Transplant to Novel Therapeutics

The authors review the potential for immunomodulatory agents in combination with cellular therapies, donor lymphocyte infusion, and following stem cell transplantation. [Leuk Lymphoma] Abstract

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

Massachusetts General Hospital and ElevateBio Announce a 10-Year Alliance Agreement to Utilize ElevateBio BaseCamp to Manufacture Innovative Cell and Gene Therapies

Massachusetts General Hospital (MGH) and ElevateBio announced that they have entered into a 10-year alliance agreement. The agreement provides MGH preferred access to ElevateBio’s BaseCamp research, process development and manufacturing facility for development and production of highly innovative cell and gene therapies developed at MGH. [ElevateBio] Press Release

Broad Institute and MilliporeSigma Announce CRISPR License Framework to Encourage Innovation

MilliporeSigma and the Broad Institute of MIT and Harvard announced an agreement to offer non-exclusive licenses to CRISPR intellectual property under their respective control for use in commercial research and product development. [Broad Institute] Press Release

IVERIC bio Announces Successful Advancement of LCA10 Minigene Program and Expansion of Orphan Gene Therapy Portfolio with USH2A-Related Inherited Retinal Diseases Program

IVERIC bio, Inc. announced that the company has exercised its option and entered into an exclusive global license agreement with the University of Massachusetts Medical School for rights to develop and commercialize mutation independent novel adeno-associated virus gene therapy product candidates for the treatment of Leber Congenital Amaurosis type 10 (LCA10) due to mutations to the CEP290 gene, the most common type of LCA. [IVERIC bio, Inc.] Press Release

Allergan and Editas Medicine Initiate the Brilliance Phase I/II Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10

Allergan plc and Editas Medicine, Inc. announced the Brilliance Phase I/II clinical trial of EDIT-101 is open for patient enrollment. AGN-151587 is an experimental medicine under development for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the CEP290 gene. [Editas Medicine, Inc.] Press Release

Abeona Therapeutics Announces Positive Interim Data from the ABO-102 Phase I/II Gene Therapy Clinical Trial in MPS IIIA

Abeona Therapeutics Inc announced positive data from its ongoing Phase I/II clinical trial evaluating ABO-102, the company’s investigational one-time, adeno-associated virus 9 gene therapy for Sanfilippo syndrome type A (MPS IIIA). [Abeona Therapeutics Inc.] Press Release

Adaptimmune Starts SPEARHEAD-1 Trial with ADP-A2M4 SPEAR T Cells for Patients with Synovial Sarcoma or MRCLS

Adaptimmune Therapeutics plc has started its SPEARHEAD-1 trial with ADP-A2M4 SPEAR T cells for patients with synovial sarcoma or myxoid/round cell liposarcoma (MRCLS). [Adaptimmune Therapeutics plc] Press Release

Orchard Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for OTL-103 for the Treatment of Wiskott-Aldrich Syndrome

Orchard Therapeutics announced that it has received RMAT designation from the FDA for OTL-103, Orchard’s ex vivo autologous hematopoietic stem cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome. [Orchard Therapeutics] Press Release

OncoSec Receives Notice of Allowance from US Patent and Trademark Office for Patent Application with Claims Covering Cytokine-Based Intratumoral Immunotherapies

OncoSec Medical Incorporated announced that the United States Patent and Trademark Office has issued a notice of allowance for patent application 15/561,915. The allowed patent application covers methods of treating a patient having treatment-refractory, cutaneous or subcutaneous, cancerous tumors, that involve injecting the tumor with a plasmid coding for an immunostimulatory cytokine, electroporating the tumor, and administering a checkpoint inhibitor to the patient. [OncoSec Medical Incorporated] Press Release

Japan Approves First Human-Animal Embryo Experiments

A Japanese stem-cell scientist is the first to receive government support to create animal embryos that contain human cells and transplant them into surrogate animals since a ban on the practice was overturned earlier this year. [Nature News] Editorial

Trump Administration Releases Details on Fetal Tissue Restrictions

The National Institutes of Health detailed how President Donald Trump’s administration will implement restrictions it announced in June on the use of human fetal tissue in research. The policy doesn’t kick in immediately, meaning grant applications already in the pipeline at NIH won’t be affected. But starting in late September, scientists applying for grants will need to explain in detail why they need to use fetal tissue and how it will be obtained. [ScienceMagazine] Editorial

Medical Journal Editors Expect Authors to Disclose Conflicts of Interest—But Don’t Disclose Their Own

Virtually all top medical journals require authors to disclose potential conflicts of interest, but few- just 12%- apply that same medicine to their own editors by publicly disclosing editors’ financial ties to industry, a study has found. [ScienceMagazine] Editorial

UK Prime Minister Boris Johnson’s Hardline Brexit Stance Stokes Fears for Scientists

Boris Johnson, a charismatic and incautious politician with scant public views on science, became U.K. prime minister last week. He immediately packed his Cabinet with ministers pledging to exit the European Union by a 31 October deadline, even without a deal in place for an amicable divorce—the “no-deal Brexit” that economists predict would cause a recession and scientists say would cause additional hardships for research. [ScienceMagazine] Editorial

NEW International Conference on Genomes & AI: From Packing to Regulation
October 2-4, 2019
Singapore, Singapore

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

NEW Scientific Communications Coordinator (STEMCELL Technologies Inc.)

Postdoctoral Scientist – HSV Vectors as a Therapy for Nervous System Tumors (Nationwide Children’s Hospital)

Senior Research Associate – Cell & Gene Therapy (King’s College London)

Postdoctoral Position – Translational Gene Therapy (Fred Hutchinson Cancer Research Center)

Senior Scientist – Immuno-Oncology of Hematologic Malignancies (University of Kansas School of Medicine)

Research Scientist – Cellular Electrophysiology (Duke University)

Postdoctoral Scientist – Computational Biologist (Cedars-Sinai Medical Center)

Senior Scientist – Antigen Presentation (Astellas Pharma)

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

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

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