Cell Therapy News Volume 19.17 | May 14 2018

TOP STORY

Survival of Syngeneic and Allogeneic iPSC-Derived Neural Precursors after Spinal Grafting in Minipigs

Scientists demonstrated that syngeneic porcine induced pluripotent stem cell (iPSC)-derived neural precursor cell transplantation to the spinal cord in the absence of immunosuppression is associated with long-term survival and neuronal and glial differentiation. [Sci Transl Med] Abstract | Press Release

(Ranked by impact factor of the journal)

CRISPR-LbCpf1 Prevents Choroidal Neovascularization in a Mouse Model of Age-Related Macular Degeneration

Unlike aflibercept, LbCpf1 targeted to vascular endothelial growth factor A or hypoxia inducing factor 1a achieved a long-term therapeutic effect on choroidal neovascularizations, potentially avoiding repetitive injections. [Nat Commun] Full Article

Self-Condensation Culture Enables Vascularization of Tissue Fragments for Efficient Therapeutic Transplantation

The self-condensation of human and mouse islets with endothelial cells not only promoted functionalization in culture but also massively improved post-transplant engraftment. Therapeutically, fulminant diabetic mice were more efficiently treated by a vascularized islet transplant compared with the conventional approach. [Cell Rep] Full Article | Press Release | Graphical Abstract

CXCL12/CXCR4 Signaling Enhances Human PSC-Derived Hematopoietic Progenitor Function and Overcomes Early In Vivo Transplantation Failure

Scientists quantified a failure of human pluripotent stem cell (hPSC)-hematopoietic progenitor cells (HPCs) to survive even 24 hour post transplantation. Across several hPSC-HPC differentiation methodologies, they identified the lack of CXCR4 expression and function. [Stem Cell Reports] Abstract | Graphical Abstract

PiggyBac-Engineered T-Cells Expressing CD19-Specific CARs that Lack IgG1 Fc Spacers Have Potent Activity against B-ALL Xenografts

Researchers designed three CD19-specifc CARs that lacked the IgG1 Fc region, and incorporated combinations of CD28 or 4-1BB transmembrane and co-stimulatory domains. At high doses, CAR19 T-cells were effective against B-ALL in a xenograft mouse model, regardless of co-stimulatory domain. [Mol Ther] Abstract

Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia

Investigators report a new conditional mouse model with complete frataxin deletion in parvalbumin positive cells which recapitulate the sensory ataxia and neuropathy associated to Friedreich Ataxia, albeit with a more rapid and severe course. Interestingly, proprioceptive neurons survived for many weeks without frataxin, although fully dysfunctional. [Mol Ther] Abstract

Overexpression of Cx43 in Cells of the Myocardial Scar: Correction of Post-Infarct Arrhythmias through Heterotypic Cell-Cell Coupling

The authors tested lentivirus-mediated delivery of the gap-junction protein connexin 43 (Cx43) into acute myocardial lesions. Cx43 was expressed in (myo)fibroblasts and CD45⁺ cells within the scar and provided prominent and long lasting arrhythmia protection in vivo. [Sci Rep] Full Article | Press Release

CAR T Cell Therapy for Breast Cancer: Harnessing the Tumor Milieu to Drive T Cell Activation

First and second generation mucin1-specific CARs were constructed using the HMFG2 scFv. In vivo anti-tumor activity was assessed using IL4-producing MDA MB 468 tumor-bearing mice using calipers to assess tumor volume and bioluminescence imaging to track T cells. [J Immunother Cancer] Full Article

Regulated Intratumoral Expression of IL-12 Using a RheoSwitch Therapeutic System^® (RTS^®) Gene Switch as Gene Therapy for the Treatment of Glioma

The authors determined if localized delivery of IL-12 encoded by a replication-incompetent adenoviral vector engineered to express IL-12 via a RTS^® gene switch administered intratumorally which is inducibly controlled by the oral activator veledimex is an effective approach for glioma therapy. [Cancer Gene Ther] Abstract

Endostatin Gene Therapy Delivered by Attenuated Salmonella typhimurium in Murine Tumor Models

Researchers constructed a S. typhimurium mutant named S634 harboring aroA mutation and additional mutations involved in modifications of lipid A. Upon intraperitoneal infection in mice, the aroA-deficient strain S634 showed greatly attenuated virulence and preferential accumulation within tumor tissue. [Cancer Gene Ther] Abstract

Cell Therapy Trials for Heart Regeneration — Lessons Learned and Future Directions

The effects of cell therapy on heart regeneration in patients with chronic cardiomyopathy have been assessed in several clinical trials. These trials can be categorized as those using noncardiac stem cells, including mesenchymal stem cells, and those using cardiac-committed cells, including KIT⁺ cardiac stem cells, cardiosphere-derived cells, and cardiovascular progenitor cells derived from embryonic stem cells. [Nat Rev Cardiol] Abstract

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Celyad Announces Presentations

Celyad announced that the company will present recent advances in Celyad’s pipeline. [Press release from Celyad discussing research to be presented at the 2018 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Chicago] Press Release

Precision BioSciences to Present In Vivo Gene Therapy and Ex Vivo Cancer Immunotherapy Data

Precision BioSciences will give three presentations at an upcoming meeting. [Press release from Precision BioSciences discussing research to be presented at the 2018 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Chicago] Press Release

Atara Biotherapeutics Expands T-Cell Immunotherapy Collaboration to Advance Next-Generation CAR T Technologies in Oncology, Autoimmune and Other Diseases

Atara Biotherapeutics, Inc. announced the company has expanded its collaboration with Memorial Sloan Kettering Cancer Center to develop the next generation of genetically engineered CAR T immunotherapies. [Atara Biotherapeutics] Press Release

RXi Pharmaceuticals Enters into Material Transfer Agreement with Iovance Biotherapeutics to Study Its Self-Delivering RNAi Technology for the Advancement of Immuno-Oncology Therapeutics with Iovance’s Tumor Infiltrating Lymphocytes (TILs) for Solid Tumors

RXi Pharmaceuticals Corporation announced that it has entered into a research collaboration with Iovance Biotherapeutics to evaluate the potential synergies with RXi’s novel sd-rxRNA therapeutic compounds and Iovance’s autologous cell therapy based on TILs for the use in the treatment of cancer. [RXi Pharmaceuticals] Press Release

Arrowhead Doses First Hepatitis B Patients in Multiple Dose Portion of Phase I/II Study of ARO-HBV

Arrowhead Pharmaceuticals Inc. announced that it has dosed the first three hepatitis B virus (HBV) patients in the multiple-ascending dose portion of its ongoing Phase I/II study of ARO-HBV, the company’s third generation subcutaneously administered RNA interference therapeutic being developed as a potentially curative therapy for patients with chronic HBV infection. [Arrowhead Pharmaceuticals Inc.] Press Release

Medigene Significantly Expands TCR Alliance with bluebird bio in Cancer Immunotherapy

Medigene AG announced the significant expansion of its successful strategic alliance with bluebird bio focusing on the research and development of T cell receptor-modified T cell (TCR-T) immunotherapies for the treatment of cancer. [Medigene AG] Press Release

Cancer-Killing Viruses Show Promise — and Draw Billion-Dollar Investment

Pharmaceutical giant Johnson & Johnson announced on 2 May that it would pay up to US$1 billion to acquire a company that makes cancer-killing viruses. It was a striking show of support for a still-unproven treatment, but the bid is just the latest sign that industry and academics are warming to the approach. [Nature News] Editorial

LogicBio Therapeutics Launches Development Program in Methylmalonic Acidemia (MMA)

LogicBio Therapeutics, Inc. announced it has initiated a development program targeting MMA—a rare, life-threatening pediatric metabolic disease. [LogicBio Therapeutics, Inc.] Press Release

PharmaCyte Biotech Successfully Completes Pore Size Studies in Cell-in-a-Box^® Capsules Used in Pancreatic Cancer Therapy

PharmaCyte Biotech, Inc. announced that it has successfully completed the “pore size studies” on its Cell-in-a Box^® capsules that are required by the FDA. [PharmaCyte Biotech, Inc.] Press Release

FDA Seeks Permanent Injunctions against Two Stem Cell Clinics

The FDA, in two complaints filed in federal court, is seeking permanent injunctions to stop two stem cell clinics from marketing stem cell products without FDA approval and for significant deviations from current good manufacturing practice requirements. [U.S. Food and Drug Administration] Press Release

Hungarian Scientists Are on Edge As Country Is Poised to Force Out Top University

In early April, several days after Viktor Orbán secured his third consecutive term—and fourth overall—as Hungarian prime minister with a landslide victory for his conservative party, the pro-government paper Figyelő; published a list of more than 200 people it called “mercenaries” of George Soros, the American-Hungarian billionaire philanthropist. The list included investigative journalists and human rights advocates—and 30 academics from the Soros-founded, Budapest-based Central European University. [ScienceInsider] Editorial

Release of Three Americans by North Korea Bolsters Hopes for Academic Diplomacy

The release of three Americans by North Korea is being welcomed in the United States. Leaders of the Pyongyang University of Science and Technology in North Korea also see the move as good news, and are hoping it might ultimately lead to the end of a travel ban that has prevented its U.S. instructors from teaching classes. [ScienceInsider] Editorial

Australian Budget Delivers for Science Facilities and Medical Research

The government will push to invest almost Aus$1.9 billion (US$1.4 billion) over the next 12 years in shared research infrastructure — such as microscopes, supercomputers, a marine observing system and telescopes used in myriad disciplines, from nanotechnology to oceanography. Medical research will receive an increase of $1.3 billion, to be spent on various programs and initiatives over 10 years that improve health and boost the medical industry, including $500 million for a genomics and precision-medicine initiative. [Nature News] Editorial

Animal Tests Surge under New U.S. Chemical Safety Law

The cause of the increase isn’t clear. But the new law imposes stricter requirements on a broader array of chemicals than its predecessor, including both new products and ones already on the market, and experts say Environmental Protection Agency staff may be trying to comply by gathering more test data from companies. [ScienceInsider] Editorial

NEW Spanish National Cancer Research Centre (CNIO): Frontiers in Immunomodulation and Cancer Therapy
July 9-11, 2018
Madrid, Spain

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NEW Postdoctoral Fellow – Gene Editing and Cellular Reprogramming (Houston Methodist Research Institute)

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

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

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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