Cell Therapy News Volume 20.04 | Feb 4 2019

TOP STORY

Self-Assembled Peptide-Poloxamine Nanoparticles Enable In Vitro and In Vivo Genome Restoration for Cystic Fibrosis

The authors showed that peptides developed by modular design approaches could spontaneously form compact and monodisperse nanoparticles with poloxamines and nucleic acids via self-assembly. Both messenger RNA and plasmid DNA expression mediated by peptide-poloxamine nanoparticles were greatly boosted in vitro and in the lungs of cystic fibrosis mice with negligible toxicity. [Nat Nanotechnol] Abstract

(Ranked by impact factor of the journal)

Endothelial Protein C Receptor Supports Hematopoietic Stem Cell Engraftment and Expansion in Mpl-Deficient Mice

By transplantation of lentiviral transduced hematopoietic cells in the Mpl-deficient mouse model scientists addressed whether known or predicted thrombopoietin target genes were able to rescue the Mpl-deficient phenotype of the mice. [Blood] Abstract | Press Release

Next-Generation Muscle-Directed Gene Therapy by In Silico Vector Design

Researchers identified potent muscle-specific transcriptional cis-regulatory modules (CRMs), containing clusters of transcription factor binding sites, using a genome-wide data-mining strategy. These novel muscle-specific CRMs resulted in a substantial increase in muscle-specific gene transcription when delivered using adeno-associated viral vectors in mice. [Nat Commun] Full Article

Operational Immune Tolerance towards Transplanted Allogeneic Pancreatic Islets in Mice and a Non-Human Primate

Investigators used a previously established approach of islet transplant in the anterior chamber of the eye in allogeneic recipient mouse models and a baboon model of diabetes, which were treated transiently with anti-CD154/CD40L blocking antibody in the peri-transplant period. [Diabetologia] Abstract | Press Release

Periodontal Regeneration by Allogeneic Transplantation of Adipose Tissue Derived Multi-Lineage Progenitor Stem Cells In Vivo

Researchers evaluated the efficacy of allogeneic transplantation of adipose-derived multi-lineage progenitor cells in a micro-mini pig periodontal defect model. [Sci Rep] Full Article

Potential Long-Term Treatment of Hemophilia A by Neonatal Co-Transplantation of Cord Blood-Derived Andothelial Colony-Forming Cells and Placental Mesenchymal Stromal Cells

The authors sought to determine whether co-transplantation of endothelial colony-forming cells and placenta-derived mesenchymal stromal cells could achieve long-term engraftment and Factor VIII expression. [Stem Cell Res Ther] Full Article

Measles Vector as a Multiple Genes Delivery Platform Facilitating iPSC Reprogramming

Scientists present a highly efficient multi-transgene delivery system based on a vaccine strain of measles virus, a non-integrating RNA virus that has a long-standing safety record in humans. [Gene Ther] Abstract

Helper-Dependent Adenovirus-Mediated Gene Transfer of a Secreted LDL Receptor/Transferrin Chimeric Protein Reduces Aortic Atherosclerosis in LDL Receptor-Deficient Mice

The authors evaluated the efficacy of the LDL receptor (LDLR)-TF chimeric protein in CHOLDLA7, a cell line lacking LDLR expression, restoring the ability to uptake LDL. Subsequently, they administered intravenously 1 × 10E13 vp/kg of this vector in LDLR-deficient mice and observed amelioration of lipid profile and reduction of aortic atherosclerosis. [Gene Ther] Abstract

Melatonin Preconditioning of Bone Marrow-Derived Mesenchymal Stem Cells Promotes their Engraftment and Improves Renal Regeneration in a Rat Model of Chronic Kidney Disease

Investigators evaluated the effect of pretreating bone marrow-derived mesenchymal stem cells with melatonin on the prosurvival and renoprotective of transplanted cells into the irreversible model of unilateral ureteral obstruction. [J Mol Histol] Abstract

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Adeno-Associated Virus Vector as a Platform for Gene Therapy Delivery

Preclinical and clinical successes in adeno-associated virus (AAV)-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. [Nat Rev Drug Discov] Abstract

Exosomes — Beyond Stem Cells for Restorative Therapy in Stroke and Neurological Injury

The authors discuss the current knowledge of exosomes and advances in our knowledge of their effects on endogenous neurovascular remodeling events. They also consider the opportunities for exosome-based approaches to therapeutic amplification of brain repair and improvement of recovery after stroke, traumatic brain injury and other diseases in which neurorestoration could be a viable treatment strategy. [Nat Rev Neurol] Abstract

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

Spark Therapeutics Announces Presentation of Preclinical Data in Pompe Disease and CLN2 Disease

Spark Therapeutics announced the presentation of preclinical data for SPK-3006 for Pompe disease and SPK-1001 for CLN2 disease, a form of Batten disease. [Press release from Spark Therapeutics discussing research to be presented at the 15^th Annual WORLDSymposium™, Orlando] Press Release

Abeona Therapeutics to Present New Supportive Data for Novel Gene Therapies

Abeona Therapeutics Inc. announced that data from ongoing clinical trials for lysosomal storage diseases and novel AIM™ chimeric AAV-based vector gene therapy programs will be presented. [Press release from Abeona Therapeutics Inc. discussing research to be presented at the 15^th Annual WORLDSymposium™, Orlando] Press Release

Neurocrine Biosciences and Voyager Therapeutics Form Strategic Development and Commercialization Collaboration for Parkinson’s Disease and Friedreich’s Ataxia

Neurocrine Biosciences, Inc. and Voyager Therapeutics, Inc. announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. [Neurocrine Biosciences, Inc.] Press Release

Janssen Enters Worldwide Collaboration and License Agreement with MeiraGTx to Develop Gene Therapy Programs for Inherited Retinal Diseases

The Janssen Pharmaceutical Companies of Johnson & Johnson announced a worldwide collaboration and license agreement with MeiraGTx Holdings plc to develop, manufacture and commercialize its clinical stage inherited retinal disease portfolio, including leading product candidates for achromatopsia caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa. [Janssen Global Services, LLC] Press Release

GenSight Biologics Reports Topline Results at Week 48 of the RESCUE Phase III Clinical Trial of GS010 in Subjects within Six Months of Visual Loss Onset Due to Leber Hereditary Optic Neuropathy (LHON)

GenSight Biologics announced results from the first scheduled readout, at week 48, of the RESCUE Phase III clinical trial evaluating the safety and efficacy of a single intravitreal injection of GS010 in 39 subjects whose visual loss due to 11778-ND4 LHON occurred up to six months prior to study treatment. [GenSight Biologics] Press Release

uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

uniQure N.V. announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release

Audentes Therapeutics Announces Next Steps in the Development of AT132 to Treat XLMTM following Collaborative Initial Meeting with FDA under RMAT Designation

Audentes Therapeutics, Inc. provided an update following the receipt of minutes from its Regenerative Medicine and Advanced Therapy (RMAT)/Type B meeting with the FDA held in December 2018, regarding the company’s lead gene therapy candidate, AT132, for the treatment of X-linked myotubular myopathy (XLMTM). [Audentes Therapeutics, Inc.] Press Release

REGENXBIO Receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease

REGENXBIO Inc. announced the FDA granted Rare Pediatric Disease Designation to RGX-181. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 gene. [REGENXBIO Inc.] Press Release

Hemostemix Announces Submission of Orphan Drug Designation Application to the FDA

Hemostemix Inc. announced that it has submitted an application with the FDA to receive Orphan Drug Designation status for its lead product ACP-01 for the treatment of critical limb ischemia. [Hemostemix Inc.] Press Release

CARsgen Therapeutics Receives IND Clearance for GPC3-CAR-T Cells from the NMPA

CARsgen Therapeutics announced that one of its leading drug candidates, humanized GPC3-CAR-T cell for the treatment of patients suffering from GPC3-positive solid tumors, has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA). [CARsgen Therapeutics (PR Newswire Association LLC.)] Press Release

Hitachi Chemical Co. Ltd. to Acquire apceth Biopharma GmbH, Expanding Global Footprint of Its Cell and Gene Therapy Contract Development and Manufacturing Services

Hitachi Chemical Advanced Therapeutics Solutions, LLC announced an agreement by which apceth Biopharma GmbH will join Hitachi Chemical’s PCT global services platform. [Hitachi Chemical Advanced Therapeutics Solutions, LLC (Business Wire, Inc.)] Press Release

First US Patient in Novel Stem Cell Trial for Stroke Disability Enrolled at UTHealth

The first U.S. patient to participate in a global study of a stem cell therapy injected directly into the brain to treat stroke disability was enrolled in the clinical trial this week at The University of Texas Health Science Center at Houston (UTHealth). [University of Texas Health Science Center at Houston] Press Release

After Shutdown, NSF Catching Up on Review Panels, Funding Requests

When the US government was partially shut down for 35 days starting December 22, work at several government agencies ground to a halt, with a total of 800,000 federal employees furloughed. The National Science Foundation (NSF), one of the sidelined science agencies, is now addressing all that went undone during that time, NSF officials told reporters. [The Scientist] Editorial

Rookies Lead the Way on House Science Panel

A major perk of being the majority party in the U.S. Congress is getting to fill the leadership slots on every committee. For several new Democratic legislators, however, having their party regain control of the House of Representatives also creates an unprecedented opportunity to shape U.S. science policy. [ScienceInsider] Editorial

Indian Government to Boost Stipend for Early-Career Scientists

The Indian government announced that it will increase the stipend for PhD students who have qualified for a research fellowship by up to 25%. [Nature News] Editorial

NEW Nature Conference on Cellular Metabolism
April 8-11, 2019
Xiamen, China

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

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

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

Cell Manufacturing Scientist (CSL Behring)

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

Postdoctoral Fellow – Inherited Retinal Degenerations (Ocular Genomics Institute)

Postdoctoral Position – Pluripotent Stem Cells and Rare Diseases (I-STEM)

Postdoctoral Fellow – Genome and Metabolic Engineering (Columbia University Medical Center)

Postdoctoral Research Associate – Gene Therapy (University of Massachusetts)

Research Fellow – Stem Cell Biology (Brigham & Women’s Hospital – Harvard Medical School)

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

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

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

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