Cell Therapy News Volume 20.15 | Apr 29 2019

TOP STORY

Allogeneic Hematopoietic Cell Transplantation Improves Outcome of Adults with T(6;9) Acute Myeloid Leukemia – Results from an International Collaborative Study

For patients undergoing allogeneic hematopoietic cell transplantation, overall survival rates at five years did not differ whether performed in first, or second complete remission or with active disease/relapse. [Haematologica] Full Article

(Ranked by impact factor of the journal)

Significantly Enhanced Recovery of Acute Liver Failure by Liver Targeted Delivery of Stem Cells via Heparin Functionalization

The authors demonstrated that enhanced delivery of human adipose-derived stem cells to a damaged liver by the coating of lipid-conjugated heparin could result in significantly improved recovery from acute liver failure in a mouse model. [Biomaterials] Abstract

On-Site Fabrication of Bi-Layered Adhesive Mesenchymal Stromal Cell-Dressings for the Treatment of Heart Failure

Scientists revealed that fibrin improved the potential of integrated mesenchymal stromal/stem cells (MSCs) through amplifying their tissue-repair abilities and activating the Akt/PI3K self-protection pathway. Outer collagen-sheets protected the MSC-fibrin complex from abrasion by surrounding tissues and also facilitated easy handling. [Biomaterials] Full Article

Gene Editing-Mediated Disruption of Epidermolytic Ichthyosis-Associated KRT10 Alleles Restores Filament Stability in Keratinocytes

Investigators utilized transcription activator-like effector nuclease technology to disrupt disease-causing mutant KRT10 alleles in an ex vivo cellular approach, with the intent of developing a therapy for epidermolytic ichthyosis patients. [J Invest Dermatol] Abstract | Full Article | Graphical Abstract

Silk Fibroin Scaffolds Seeded with Wharton’s Jelly Mesenchymal Stem Cells Enhance Re-Epithelialization and Reduce Formation of Scar Tissue after Cutaneous Wound Healing

Scientists investigated the wound healing effects of electrospun silk fibroin scaffolds cellularized with human Wharton’s jelly mesenchymal stem cells using a murine excisional wound splinting model. [Stem Cell Res Ther] Full Article

Bone-Derived Nestin-Positive Mesenchymal Stem Cells Improve Cardiac Function via Recruiting Cardiac Endothelial Cells after Myocardial Infarction

After transplantation into mice exposed to myocardial infarction, Nestin⁺ bone-derived mesenchymal stem cell (BMSC)-treated mice showed significantly improved survival and left ventricular function compared with Nestin− BMSC-treated mice. [Stem Cell Res Ther] Full Article

Multifunctional Drug Carrier Based on PEI Derivatives Loaded with Small Interfering RNA for Therapy of Liver Cancer

In vivo imaging results showed that fluorescently labeled siRNA loaded in sTPssOLP was able to deliver more to the tumor site. At the same time, it was observed that sTPssOLP did not show significant damage to normal tissues. [Int J Pharm] Abstract | Full Article

AAV9 Gene Replacement Therapy for Respiratory Insufficiency in Very‐Long Chain Acyl‐CoA Dehydrogenase Deficiency

Researchers aimed to characterize respiratory pathophysiology in very‐long chain acyl‐CoA dehydrogenase deficient mice, and to determine if AAV9‐mediated gene therapy improves respiratory function. [J Inherit Metab Dis] Abstract

Mesenchymal Stem Cells Pretreated with Platelet-Rich Plasma Modulate Doxorubicin-Induced Cardiotoxicity

Mesenchymal stem cells (MSCs) and platelet-rich plasma (PRP)/MSCs attenuated doxorubicin-induced cardiotoxicity. Better attenuation was observed in the PRP/MSC-treated group. PRP/MSC combination reduced greatly the malondialdehyde and tumor necrosis factor-α and increased IL-10, Bcl2/Bax ratio, and vascular endothelial growth factor. [Hum Exp Toxicol] Abstract

Quantitative Systems Pharmacology Model of Chimeric Antigen Receptor T‐Cell Therapy

The quantitative systems pharmacology approach was used to quantify the complex relationships among chimeric antigen receptor T‐cell (CART) doses, disease burden, and pro inflammatory cytokines in human subjects and to gain relevant insights into the determinant of clinical toxicity/efficacy in development of CART therapy. [Clin Transl Sci] Full Article

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Delivering Cellular and Gene Therapies to Patients: Solutions for Realizing the Potential of the Next Generation of Medicine

The authors review the existing system to deliver cell and gene therapies and outlines the requirements to make them accessible to patients. Informed by interviews with experts, opportunities for improvement are identified along the patient and cell journeys, and a call to action is made for stakeholders to detail and implement change. [Gene Ther] Full Article

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

Exosomes Derived from Human Mesenchymal Stem Cells Ameliorate Autistic-Like Behaviors

Stem Cell Medicine Ltd. presented results of a study of its proprietary mesenchymal stem cell-derived exosomes for the treatment of autistic disorders. [Press release from Stem Cell Medicine Ltd. discussing research presented at the International Society for Extracellular Vesicles (ISEV) 2019 Annual Meeting, Kyoto] Press Release

Vivet Therapeutics Announces 2 Abstracts Accepted for Oral Presentation

Vivet Therapeutics announced that new data from its lead development program, VTX-801 a novel investigational gene therapy for WD, and VTX-803, Vivet’ novel therapeutic in development for the treatment of PFIC3, have been accepted for oral presentation. [Press release from Vivet Therapeutics (Business Wire, Inc.) discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Fate Therapeutics Announces Five Presentations

Fate Therapeutics, Inc. announced that Fate scientists and collaborators will present data highlighting the company’s induced pluripotent stem cell (iPSC) product platform and its iPSC-derived cell product candidates. [Press release from Fate Therapeutics, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Precision BioSciences Announces Presentations

Precision BioSciences announced its participation in several preclinical presentations and a Genome Editing Workshop presentation. [Press release from Precision BioSciences discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

SQZ Biotech to Present at the American Society of Gene and Cell Therapy 22^nd Annual Meeting

SQZ Biotechnologies, a cell therapy company developing novel treatments for multiple therapeutic areas, announced presentations. [Press release from SQZ Biotechnologies discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

MeiraGTx Announces Upcoming Presentation on Achromatopsia Gene Therapy Candidate AAV-CNGA3

MeiraGTx Holdings plc announced a pre-clinical poster on safety and efficacy of the company’s gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia due to mutations in the CNGA3 gene will be presented. [Press release from MeiraGTx Holdings plc discussing research to be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2019 Annual Meeting, Vancouver] Press Release

Magenta Therapeutics to Present Preclinical Data on E478 Stem Cell Gene Therapy Expansion Program

Magenta Therapeutics announced the company will highlight preclinical data on its E478 program in an oral presentation. [Press release from Magenta Therapeutics discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

BioRestorative Therapies to Present as to Data on its Platform Technology for the Treatment of Metabolic Disorders

BioRestorative Therapies, Inc. announced that Francisco Silva, Vice President of Research and Development, will present. [Press release from BioRestorative Therapies, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Moderna to Present Preclinical Data

Moderna, Inc. announced that it will present, along with academic collaborators, preclinical data from seven studies that support the potential of mRNA-based therapies to treat the underlying cause of several rare metabolic and genetic disorders. [Press release from Moderna, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Orchard Therapeutics Announces Clinical Proof-of-Concept Data for Gene Therapy OTL-300 Demonstrating Efficacy in Transfusion-Dependent Beta-Thalassemia

Orchard Therapeutics announced the presentation of the full clinical proof-of-concept data from its trial of ex vivo, autologous, hematopoietic stem cell gene therapy, OTL-300, for the treatment of transfusion-dependent beta-thalassemia. [Press release from Orchard Therapeutics discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Intellia Therapeutics Presents New In Vivo and Engineered Cell Therapy Data

Intellia Therapeutics, Inc. will present new data, including the first demonstration of targeted gene insertion with CRISPR/Cas9 in the liver of non-human primates. [Press release from Intellia Therapeutics, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Translate Bio Announces Oral Presentation of mRNA Therapeutics in Models of Metabolic Disorders

Translate Bio, Inc. presented preclinical results that demonstrate the potential of mRNA therapeutics as treatments for urea cycle disorders and organic acidemias. [Press release from Translate Bio, Inc. discussing research to be presented at the American Society of Gene and Cell Therapy (ASGCT) 22^nd Annual Meeting, Washington, DC] Press Release

Allergan and Editas Medicine Announce Initial Data from Natural History Study to Evaluate Patients with Leber Congenital Amaurosis 10 (LCA10)

Allergan plc and Editas Medicine, Inc. announced initial data from the ongoing natural history study to evaluate patients with LCA10, a rare form of blindness caused by mutations in the CEP290 gene. [Press release from Allergan plc (GlobeNewswire, Inc.) discussing research presented at the 6^th Annual Retinal Cell and Gene Therapy (RCGT) Innovation Summit, Vancouver] Press Release

Rexgenero Presents Update on Phase III Clinical Trials with REX-001, a Cell-Based Therapy for Critical Limb Ischaemia

Rexgenero presented an update on its two Phase III SALAMANDER trials being conducted across Europe for its lead development programme REX-001 to treat CLI in patients with diabetes. [Press release from Rexgenero (Business Wire, Inc.) discussing research presented at the Cell and Gene Therapy Meeting on the Med, Barcelona] Press Release

UC Receives Fourth CRISPR Patent; Three More on the Way

The U.S. Patent Office issued a fourth patent for the revolutionary CRISPR-Cas9 gene-editing technology to the University of California, expanding the university’s patent portfolio to cover a broad variety of uses in all types of cells as well as cell-free environments. [UC Berkeley] Press Release

TC BioPharm Initiates Phase I Trial of Allogeneic Gamma Delta T Cell Therapy in Acute Myeloid Leukemia Patients

TC BioPharm announced it has initiated a Phase I clinical study of TCB002, an allogeneic cell therapy consisting of activated and expanded gamma delta T cells. [TC BioPharm] Press Release

International Stem Cell Corporation Completes Enrollment and Dosing in Its Parkinson’s Disease Clinical Trial

International Stem Cell Corporation announced the completion of subject enrollment in its Phase I clinical trial of ISC-hpNSC^® for the treatment of Parkinson’s disease. [International Stem Cell Corporation] Press Release

Gracell Bio Announces FasT CAR-T, a Breakthrough Technology for Hematological Malignancies

Gracell Biotechnologies, Co., Ltd. announced it has developed FasT CAR-T, a revolutionary platform that shortens the manufacturing time of CAR-T treatments from two weeks to one day. [Gracell Biotechnologies, Co., Ltd. (PR Newswire Association LLC.)] Press Release

HEALIOS K.K. Announces Enrollment of First Patient in Japan in the ONE-BRIDGE Study of MultiStem^® Treatment for Acute Respiratory Distress Syndrome

Athersys, Inc. announced that its partner, HEALIOS K.K., has enrolled the first patient in its ONE-BRIDGE study in Japan, evaluating MultiStem^® cell therapy treatment of patients who suffer from acute respiratory distress syndrome. [Athersys, Inc.] Press Release

Autolus Therapeutics Receives FDA Orphan Drug Designation for AUTO3 for Treatment of Acute Lymphoblastic Leukemia

Autolus Therapeutics plc announced that the FDA has granted orphan drug designation to autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors targeting CD19 and CD22 (AUTO3) for the treatment of acute lymphoblastic leukemia. [Autolus Therapeutics plc] Press Release

Kite Announces Plans for New State-of-the-Art Facility to Expand Cell Therapy Production Capabilities

Kite announced plans for a new facility in Frederick County, Maryland, which will produce innovative cell therapies for people with cancer. [Kite (Business Wire, Inc.)] Press Release

Orchard Therapeutics Announces First Patient Dosed with Cryopreserved Formulation of OTL-103 Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

Orchard Therapeutics announced that the first patient with Wiskott-Aldrich Syndrome has been dosed in a open label study designed to evaluate engraftment of the cryopreserved formulation of OTL-103, its ex vivo autologous hematopoietic stem cell gene therapy. [Orchard Therapeutics] Press Release

AVROBIO, Inc. Announces FDA Clearance of Investigational New Drug Application for AVR-RD-01 Gene Therapy for the Treatment of Fabry Disease

AVROBIO, Inc. announced that the FDA has cleared the company’s Investigational New Drug application for AVR-RD-01, its gene therapy candidate for the treatment of Fabry disease. [AVROBIO, Inc.] Press Release

LogicBio Therapeutics Receives FDA Orphan Drug Designation for LB-001 for the Treatment of Methylmalonic Acidemia

LogicBio Therapeutics, Inc. announced that the FDA has granted orphan drug designation to LB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase gene for the treatment of methylmalonic acidemia. [LogicBio Therapeutics, Inc.] Press Release

US Universities Reassess Collaborations with Foreign Scientists in Wake of NIH Letters

Kuspa’s attempt to stay ahead of NIH came to naught. A few months into the audit, Baylor College of Medicine received letters from the US National Institutes of Health (NIH) asking about four scientists it believed had violated the agency’s rule requiring them to disclose all foreign ties relating to their research. [ScienceInsider] Editorial

Elsevier and Norway Agree on New Open-Access Deal

After unsuccessful negotiations between a coalition of Norwegian organizations and the academic publisher Elsevier culminated in cancelled subscriptions earlier this year, the two have successfully established a new nationwide licensing agreement. [The Scientist] Editorial

FDA

Determination of Regulatory Review Period for Purposes of Patent Extension; YESCARTA (FR Doc. No:2019-08609) Notice

NIH

Final Action under the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines) (FR Doc. No:2019-08462) Notice

NEW International Conference on Cellular Therapies: Focus on Immune Based Therapeutic Concepts
September 20-22, 2019
Mandelieu, France

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

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

Stem Cell Podcast Co-Host (Stem Cell Podcast)

Postdoctoral Associate – Cancer Immunology (Winship Cancer Institute)

DPhil Scholarship – Retinal Gene Therapy (University of Oxford)

Gene Editing Specialist – Rare Genetic Disorders (Novo Nordisk)

Director of Research – Division of Translational Medicine & Human Genetics (University of Pennsylvania)

Postdoctoral Research Associate – Retinal Stem Cell Biology and Regeneration (University of Nebraska Medical Center)

Cell Biologist – Regenerative Medicine (Hepatx Corporation)

Associate Scientist – Immuno-Oncology (Hackensack Meridian Health Center for Discovery & Innovation)

Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

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)

Faculty Positions – Hematology and Oncology (University of California, Davis)

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