Cell Therapy News Volume 19.14 | Apr 23 2018

TOP STORY

Editing out Blood Disease

In a powerful example of bench-to-bedside science showing how observations made in the lab can spark life-altering therapies in the clinic, an international team of investigators has announced that gene therapy can be safe and effective for patients with a severe form of the blood disorder beta-thalassemia. [Press release from Harvard Medical School discussing online prepublication in The New England Journal of Medicine] Press Release | Press Release 2 | Press Release 3 | Abstract | Editorial

(Ranked by impact factor of the journal)

Systemic Administration of the Antisense Oligonucleotide NS-065/NCNP-01 for Skipping of Exon 53 in Patients with Duchenne Muscular Dystrophy

Scientists conducted a Phase I, open-label, dose-escalation clinical trial to determine the safety, pharmacokinetics, and activity of NS-065/NCNP-01, a morpholino antisense oligonucleotides that enables skipping of exon 53. The primary endpoint was safety; the secondary endpoints were pharmacokinetics and successful exon skipping. [Sci Transl Med] Abstract

Fully Reduced HMGB1 Accelerates the Regeneration of Multiple Tissues by Transitioning Stem Cells to G_Alert

Researchers showed that high mobility group box 1 (HMGB1) is released following fracture in both humans and mice, forms a heterocomplex with CXCL12, and acts via CXCR4 to accelerate skeletal, hematopoietic, and muscle regeneration in vivo. [Proc Natl Acad Sci USA] Full Article

Strategic Therapeutic Targeting to Overcome Venetoclax Resistance in Aggressive B-Cell Lymphomas

Mantle cell lymphoma and diffuse large B cell lymphoma cell lines, primary patient samples, and in vivo patient-derived xenograft models were utilized to examine venetoclax efficacy. [Clin Cancer Res] Abstract | Full Article

Targeting the Leukemia Antigen PR1 with Immunotherapy for the Treatment of Multiple Myeloma

Scientists tested if multiple myeloma (MM) is capable of cross-presenting PR1 and subsequently becomes susceptible to PR1-targeting immunotherapies, using MM cell lines, a xenograft mouse model, and primary MM patient samples. Following PR1 cross-presentation, they were able to target MM with PR1-cytotoxic T lymphocytes and anti-PR1/human leukocyte antigen-A2 antibody both in vitro and in vivo. [Clin Cancer Res] Abstract

Allergen-Specific Immunotherapy Induces Regulatory T Cells in an Atopic Dermatitis Mouse Model

Researchers established an allergen-specific immunotherapy (SIT) model on atopic dermatitis mice and showed that their model correlates well with previous reports about SIT-treated patients. [Allergy] Abstract

Differential Impact of Transplantation on Peripheral and Tissue-Associated Viral Reservoirs: Implications for HIV Gene Therapy

The authors present a nonhuman primate model of latent infection using simian/human immunodeficiency virus and combination antiretroviral therapy in pigtail macaques. They demonstrated that transplantation of CCR5 gene-edited hematopoietic stem/progenitor cells persist in infected and suppressed animals, and that protected cells expand through virus-dependent positive selection. [PLoS Pathog] Full Article | Press Release

ROS-Inducing Micelles Sensitize Tumor-Associated Macrophages to TLR3 Stimulation for Potent Immunotherapy

Researchers developed galactose-functionalized zinc protoporphyrin IX grafted poly(L-lysine)-b-poly(ethyleneglycol) polypeptide micelles for tumor-associated macrophage(TAM)-targeted immunopotentiator delivery, which aimed at in vivo repolarization of TAMs to anti-tumor M1 macrophages. [Biomacromolecules] Abstract

Gadd45a Gene Silencing by RNAi Promotes Cell Proliferation and Inhibits Apoptosis and Senescence in Skin Squamous Cell Carcinoma through the p53 Signaling Pathway

Gadd45a silencing increased tumor cell proliferation and reduced apoptosis and senescence through the p53 signaling pathway in skin squamous cell carcinoma. Cell apoptosis and senescence were inhibited, while cell viability and proliferation were promoted after siGadd45a treatment. [J Cell Physiol] Abstract

Targeting Wnt/β-Catenin Signaling for Cancer Immunotherapy

Increasing evidence indicates that Wnt/β-catenin signaling, one of the best-characterized cancer drivers, promotes cancer progression by regulating the tumor-immune cycle in most of the nodes, including dendritic cells, T cells, and tumor cells. Scientists propose that targeting Wnt/β-catenin signaling would potentially improve clinical outcomes of cancer patients by overcoming the primary, adaptive, and acquired resistance to immunotherapy. [Trends Pharmacol Sci] Abstract

Strategies for In Vivo Genome Editing in Nondividing Cells

To bypass the problem of homology-directed repair (HDR) inefficiency in nondividing cells, HDR-independent strategies are being developed to efficiently manipulate the genomes of these cells. [Trends Biotechnol] Abstract

Pharmacological Approaches Promoting Stem Cell-Based Therapy following Ischemic Stroke Insults

The authors summarize the limitations of ischemia-induced neurogenesis and stem-cell transplantation, as well as the potential proneuroregenerative effects of drugs that may enhance efficacy of cell-based therapies. [Acta Pharmacol Sin] Full Article

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

Alnylam Presents New Positive Clinical Results for Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias

Alnylam Pharmaceuticals, Inc. announced new results from the Phase I and Phase I/II open-label extension studies of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyrias. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the European Association for the Study of the Liver (EASL) 53^rd Annual International Liver Congress^™, Paris] Press Release

Obsidian Therapeutics Presents Preclinical Data from Regulated Cytokine Programs that Enable CAR-T Therapies with Controllable Functions

Obsidian Therapeutics, Inc. announced the presentation of preclinical data on its regulated cytokine programs. [Press release from Obsidian Therapeutics, Inc. (Business Wire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Endocyte Presents Data from Its CAR T Platform

Endocyte, Inc. announced in a late-breaking poster session the presentation of new research from Endocyte’s CAR T adaptor molecule platform. [Press release from Endocyte, Inc. discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

Abramson Cancer Canter Studies Show Promise of Immunotherapy Combinations, Including CAR T

New studies are providing fresh clues on potentially effective combinations with CAR T therapy in brain cancer as well as a novel therapeutic target in head and neck cancer, and also providing greater understanding of the mechanisms of resistance in pancreatic cancer. [Press release from the University of Pennsylvania discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2018, Chicago] Press Release

MiMedx to Highlight Efficacy of Its Allografts in Presentations

MiMedx Group, Inc. announced the company’s participation at the SAWC Spring Conference. [Press release from MiMedx Group, Inc. discussing research to be presented at the 2018 Symposium on Advanced Wound Care Spring | Wound Healing Society (SAWC Spring/WHS) Meeting, Charlotte] Press Release

$10m in Funding Awarded to 11 Projects to Boost Health Innovation

MTPConnect – the Australian Government’s Medical Technology, Biotechnology, and Pharmaceutical Industry Growth Centre – and the Minister for Health, the Hon Greg Hunt MP, announced the first 11 recipients of $10 million investment from the Australian Government’s $35 million BioMedTech Horizons program. [MTPConnect] Press Release

ReNeuron Wins Grant for Retinal Cell Therapy Development

ReNeuron Group plc announced that it is the lead industry participant in a new £1.5 million grant award from the UK’s innovation agency, Innovate UK, to advance development of its hRPC stem cell therapy candidate for blindness-causing degenerative diseases of the retina. [ReNeuron Group plc] Press Release

UTHealth Receives Funding to Study Stem Cell Therapy for Traumatic Injury

The University of Texas Health Science Center at Houston (UTHealth) has received funding through a public/private partnership for the first-ever clinical trial investigating a stem cell therapy for early treatment and prevention of complications after severe traumatic injury. [The University of Texas Health Science Center at Houston] Press Release

CHOP’s Breakthrough Cancer Immunotherapy Program Receives Generous Grant from The Emily Whitehead Foundation

The Emily Whitehead Foundation presented a check totaling $250,000 to Stephan A. Grupp, MD, PhD, Director of the Cancer Immunotherapy Frontier Program, and Section Chief of the Cellular Therapy and Transplant Program at Children’s Hospital of Philadelphia (CHOP), benefitting cellular immunotherapy research at CHOP. [The Children’s Hospital of Philadelphia] Press Release

Abeona Announces FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA

Abeona Therapeutics Inc. announced that the FDA has granted Regenerative Medicine Advanced Therapy designation to ABO-102, the company’s adeno-associated virus-mediated gene therapy for the treatment of Sanfilippo syndrome Type A, a rare autosomal-recessive lysosomal storage disease. [Abeona Therapeutics Inc.] Press Release

Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease

Abeona Therapeutics Inc. announced that the European Medicines Agency Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona’s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children. [Abeona Therapeutics Inc.] Press Release

Alnylam Receives Positive Opinion for Orphan Drug Designation in the European Union for ALN-TTRsc02, a Subcutaneously Delivered Investigational RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis

Alnylam Pharmaceuticals, Inc. announced that the European Medicines Agency Committee for Orphan Medicinal Products has issued a positive opinion recommending ALN-TTRsc02, an investigational, subcutaneously administered RNAi therapeutic, for designation as an orphan medicinal product for the treatment of transthyretin-mediated amyloidosis. [Alnylam Pharmaceuticals, Inc.] Press Release

New Stem Cell Therapy May Transform Heart Failure Treatment

Clinical trial planning is underway at MedStar Heart & Vascular Institute to determine whether a novel stem cell therapy will improve heart function for patients with heart failure. MedStar Heart, in partnership with CardioCell, a subsidiary of Stemedica Cell Technologies, pioneered the use of stem cells in regenerative medicine. The trial will use CardioCell’s proprietary mesenchymal stem cells, manufactured by Stemedica. [Stemedica Cell Technologies, Inc.] Press Release

Cynata Therapeutics Completes Patent Application to Cover Cymerus^™ Stem Cell Technology in the Treatment of Side Effects Related to CAR-T Therapy

Cynata Therapeutics Limited announced that it has completed the filing of a patent application with IP Australia that would cover the therapeutic use of its Cymerus^™ stem cell technology in the treatment of adverse reactions associated with CAR-T immunotherapy. [Cynata Therapeutics (GlobeNewswire, Inc.)] Press Release

Penn Medicine’s Carl June, MD, Named One of Time Magazine’s Most Influential People in the World

TIME named University of Pennsylvania cancer and HIV gene therapy pioneer Carl June, MD, to the 2018 TIME 100, its annual list of the 100 most influential people in the world. The list, now in its fifteenth year, recognizes the activism, innovation and achievement of the world’s most influential individuals, and is honoring June for his pioneering work in developing CAR T therapy, which became the nation’s first FDA-approved personalized cellular therapy for cancer in August 2017. [University of Pennsylvania] Press Release

Cell and Gene Therapy Catapult Opens Manufacturing Centre to Accelerate Growth of the Industry in the UK

Cell and Gene Therapy Catapult’s unique cell and gene therapy manufacturing center was officially opened by the Business Secretary Greg Clark and Science Minister Sam Gyimah. Backed by over £60 million of UK Government investment from its Industrial Strategy, the centre will support and develop the rapidly growing global cell and gene therapy industry in the United Kingdom whilst demonstrating the government’s modern Industrial Strategy in action. [Cell and Gene Therapy Catapult] Press Release

KEI Sues NIH over License of CD30 CAR T Patents to Gilead

Knowledge Ecology International (KEI) filed a lawsuit against the National Institutes of Health (NIH) to block or invalidate an exclusive license of patents on a new CAR T therapy to Gilead Sciences. [Knowledge Ecology International] Editorial

Salk Puts Cancer Scientist Inder Verma on Leave after Harassment Allegations, Announces Investigation

The Salk Institute for Biological Studies has placed prominent cancer and gene therapy scientist Inder Verma on administrative leave and launched an investigation into allegations against him. [ScienceInsider] Editorial

Women Less Successful When Applying for Patents

Women inventors have less success than men at each step of the patent application process in the U.S., according to a study published this month in Nature Biotechnology. [The Scientist] Editorial

NIH

National Center for Advancing Translational Sciences; Notice of Closed Meeting (FR Doc. No:2018-07753) Notice

NEW 3D Cell Culture 2018
June 5-7, 2018
Freiburg, Germany

NEW 11^th Annual Business of Regenerative Medicine: Innovation, Clinical Translation, and Entrepreneurship Symposium
July 17-18, 2018
Philadelphia, Pennsylvania, United States

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

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

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

NEW PhD Student – Immunology of Cell Death & Cancer Immunotherapy (KU Leuven)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

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

Postdoctoral Associate – Cancer Therapy (St. Jude Children’s Research Hospital)

Scientist – Discovery Biology (Prevail Therapeutics)

Postdoctoral Research Fellow – Cell/Gene Therapy (Stanford University)

Postdoctoral Fellow – Precision Therapy of Aggressive Lymphoma (AstraZeneca)

Postdoctoral Fellow – Leukemia (Necker Institut Enfants Malades)

Assistant Professor – Translation of Preclinical Regenerative Medicine into Advanced Therapy Medicinal Products (Lund University)

Postdoctoral Research Fellow – Hematopoiesis (Fred Hutchinson Cancer Research Center)

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

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