Cell Therapy News Volume 20.28 | Aug 19 2019

TOP STORY

De Novo Mutations in Mitochondrial DNA of iPSCs Produce Immunogenic Neoepitopes in Mice and Humans

Investigators present evidence in mice and humans that nonsynonymous mitochondrial DNA mutations can arise and become enriched during reprogramming to the induced pluripotent stem cell (iPSC) stage, long-term culture and differentiation into target cells. [Nat Biotechnol] Abstract | Press Release

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T-Cell Receptor Gene Therapy for Human Papillomavirus-Associated Epithelial Cancers: A First-in-Human, Phase I/II Study

This Phase I/II, single-center trial enrolled patients with metastatic HPV16-positive cancer from any primary tumor site who had received prior platinum-based therapy. Treatment consisted of autologous genetically engineered T cells expressing a T-cell receptor directed against HPV16 E6, a conditioning regimen, and systemic aldesleukin. [J Clin Oncol] Abstract

Regulatable Interleukin-12 Gene Therapy in Patients with Recurrent High-Grade Glioma: Results of a Phase I Trial

A multicenter Phase I dose-escalation trial treated 31 patients undergoing resection of recurrent high-grade glioma. Resection cavity walls were injected with a fixed dose of the human interleukin-12 (hIL-12) vector. The oral activator for hIL-12, veledimex, was administered preoperatively and postoperatively. [Sci Transl Med] Abstract | Press Release

AAV-ie Enables Safe and Efficient Gene Transfer to Inner Ear Cells

Scientists showed that adeno-associated virus inner ear (AAV-ie) transduced the cochlear supporting cells (SCs) with high efficiency, representing a vast improvement over conventional AAV serotypes. After AAV-ie-mediated transfer of the Atoh1 gene, they found that many SCs trans-differentiated into new hair cells. [Nat Commun] Full Article

Enhanced Adaptive Immune Responses in Lung Adenocarcinoma through Natural Killer Cell Stimulation

After stimulation, natural killer (NK) cells localized inside tumors, with increased cytokine production capacity. T cells were also recruited to tumors in an NK cell-dependent manner, and exhibited higher functionality. In neoantigen-expressing tumors, NK cell stimulation enhanced the number and function of tumor-specific T cells and, in long-term settings, reduced tumor growth. [Proc Natl Acad Sci USA] Full Article

Targeted Integration and High-Level Transgene Expression in AAVS1-Transgenic Mice after In Vivo Hematopoietic Stem Cell Transduction with HDAd5/35++ Vectors

Ex vivo and in vivo hematopoietic stem cell (HSC) transduction/selection studies with HDAd-CRISPR + HDAd-globin-donor resulted in stable γ-globin expression at levels that were significantly higher than those achieved in previous studies with a SB100x-transposase-based HDAd5/35++ system that mediated random integration. [Mol Ther] Abstract | Graphical Abstract

Targeted Migration of Human Adipose-Derived Stem Cells to Secondary Lymphoid Organs Enhances Their Immunomodulatory Effect and Prolongs the Survival of Allo‐Grafted Vascularized Composite

Intravenously infused human adipose derived stem cells (hASCs)/chemokine receptor 7 (CCR7) targetly relocated in the T cell aggression area in secondary lymphoid organs (SLOs). In a rat vascularized composite allotransplantation model, hASCs/GFP transfusion had rare effect on the allo-grafted vascularized composite. However, hASCs/CCR7 infusion potently prolonged the grafts’ survival time. [Stem Cells] Abstract

In Utero Amniotic Fluid Stem Cell Therapy Protects against Myelomeningocele via Spinal Cord Coverage and Hepatocyte Growth Factor Secretion

Scientists investigated the therapeutic effects and mechanisms of human amniotic fluid stem cell (hAFSC)-based treatment for fetal myelomeningocele. hAFSCs were isolated as CD117-positive cells from the amniotic fluid of 15- to 17-week pregnant women who underwent amniocentesis for prenatal diagnosis and consented to this study. [Stem Cells Transl Med] Full Article

Cardiac Regeneration Using Human iPSC-Derived Biomaterial-Free 3D Bioprinted Cardiac Patch In Vivo

The cardiac patches were generated using 3D bioprinting technology in conjunction with cellular spheroids created from a co-culture of human IPSC derived cardiomyocytes, fibroblasts, and endothelial cells. Once printed and cultured, the cardiac patches were implanted into a rat myocardial infarction model. [J Tissue Eng Regen Med] Abstract

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Engineering Universal Cells that Evade Immune Detection

With the advent of pluripotent stem cells, CRISPR-Cas9 and other gene-editing technologies, the race to create ‘off-the-shelf’ donor cells that are invisible to the immune system has started. One important approach for creating such cells involves the manipulation of genes required for immune recognition, in particular HLA class I and II proteins. [Nat Rev Immunol] Abstract

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Phio Pharmaceuticals and Helmholtz Zentrum München to Collaborate on Novel Targets for the Use of Self-Delivering RNAi In T Cell and NK Cell Adoptive Cell Therapy Therapeutics

Phio Pharmaceuticals Corp. announced that is has entered into a research collaboration with the Helmholtz Zentrum München. The agreement covers the design and development of new targets based on Phio Pharmaceuticals’ proprietary self-delivering RNAi platform for use in cancer immunotherapies. [Phio Pharmaceuticals Corp.] Press Release

Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome

Ultragenyx Pharmaceutical Inc. and GeneTx Biotherapeutics LLC announced a partnership to develop GeneTx’s GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. [Ultragenyx Pharmaceutical Inc.] Press Release

Inovio Completes Enrollment of its VGX-3100 Open-Label Phase II Trial for Treatment of HPV-Related High-Grade Anal Dysplasia

Inovio Pharmaceuticals, Inc. announced that it has completed enrollment in the company’s open-label, 24 patient, Phase II trial with its lead immunotherapy product VGX-3100 in patients with precancerous lesions of the anus, i.e. anal high-grade squamous intraepithelial lesions. [Inovio Pharmaceuticals, Inc.] Press Release

NKMax America Doses First Subject in Phase I Trial of SNK in Patients with Refractory Cancer

NKMax America announced that it has dosed the first subject in study SNK01-US01, a Phase I clinical trial evaluating SNK, an ex vivo expanded autologous natural killer cell therapeutic, in patients with pathologically confirmed cancer refractory to conventional therapy. [NKMax America (PR Newswire Association LLC.)] Press Release

City of Hope Now Enrolling Patients for First-in-Human T Cell Trial for HPV-Associated Cancers

The first-in-human, Phase I T cell trial for patients who have human papillomavirus (HPV)-associated cancers that have relapsed, or are resistant to treatment, is now open at City of Hope. [City of Hope] Press Release

Mustang Bio Announces $9.28 Million Grant to Fund Phase I Trial of CAR T Therapy MB-103 in HER2-Positive Breast Cancer with Brain Metastases

Mustang Bio, Inc. announced that City of Hope has received a $9.28 million grant from the California Institute for Regenerative Medicine (CIRM) to fund an ongoing Phase I clinical trial of MB-103 for the treatment of HER2-positive breast cancer with brain metastases. [Mustang Bio, Inc.] Press Release

Lineage Cell Therapeutics Awarded NIH Grant for Innovative Vision Restoration Program

Lineage Cell Therapeutics, Inc. announced that it has been awarded a new $670,621 Small Business Innovation Research grant to advance its Vision Restoration Program, the company’s proprietary and innovative program generating 3D human retinal tissue derived from pluripotent cells. [Lineage Cell Therapeutics, Inc.] Press Release

Fate Therapeutics Announces Issuance of Foundational US Patent Covering iPSC-derived CAR T Cells

Fate Therapeutics, Inc. announced that the US Patent and Trademark Office has issued US Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an induced pluripotent stem cell (iPSC). [Fate Therapeutics, Inc.] Press Release

Hundreds of Extreme Self-Citing Scientists Revealed in New Database

A data set, which lists around 100,000 researchers, shows that at least 250 scientists have amassed more than 50% of their citations from themselves or their co-authors, while the median self-citation rate is 12.7%. The study could help to flag potential extreme self-promoters, and possibly ‘citation farms’, in which clusters of scientists massively cite each other, say the researchers. [Nature News] Editorial

In Departure for NIH, Cancer Moonshot Requires Grantees to Make Papers Immediately Free

In the United States, federal agencies have stuck to a decade-old policy that allows grantees to publish in journals that keep papers behind a paywall for up to one year. Now, the divide is starting to blur, with one prominent US research program starting to require immediate open access to the peer-reviewed publications it funds. [ScienceInsider] Editorial

MHRA

Clinical Trials for Medicines: Authorization Assessment Performance Notice

NEW 7^th International Conference on Stem Cell Engineering 2019
December 11-13, 2019
Barcelona, Spain

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NEW Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

Scientific Communications Coordinator (STEMCELL Technologies Inc.)

Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University)

Postdoctoral Scholar – Gene and Cell Therapy (Stanford University)

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

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 Fellow – Single Cell Genomics (BC Cancer Research Center)

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

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

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