Cell Therapy News Volume 20.03 | Jan 28 2019

TOP STORY

UC San Diego Researchers First to Use CRISPR/Cas9 to Control Genetic Inheritance in Mice

Biologists at the University of California (UC), San Diego have developed the world’s first CRISPR/Cas9-based approach to control genetic inheritance in a mammal. [Press release from University of California, San Diego discussing online prepublication in Nature] Press Release | Abstract

(Ranked by impact factor of the journal)

AAV2.7m8 Is a Powerful Viral Vector for Inner Ear Gene Therapy

Researchers examined the infection patterns of two synthetic adeno-associated viruses in the mouse inner ear. AAV2.7m8 infected both inner hair cells and outer hair cells with high efficiency. [Nat Commun] Full Article

Improved TMC1 Gene Therapy Restores Hearing and Balance in Mice with Genetic Inner Ear Disorders

The authors report restoration of function in inner and outer hair cells, enhanced hair cell survival, restoration of cochlear and vestibular function, restoration of neural responses in auditory cortex and recovery of behavioral responses to auditory and vestibular stimulation. [Nat Commun] Full Article

Human Double Negative T Cells Target Lung Cancer via Ligand-Dependent Mechanisms that Can Be Enhanced by IL-15

Scientists demonstrated that ex vivo expanded double negative T cells could effectively lyse various human non-small-cell lung cancer cells in vitro and inhibit tumor growth in xenograft models. [J Immunother Cancer] Full Article

Cardiac and Skeletal Muscle Effects in the Randomized HOPE-Duchenne Trial

Investigators assessed the feasibility, safety, and efficacy of intracoronary allogeneic cardiosphere-derived cells in patients with Duchenne muscular dystrophy. [Neurology] Abstract | Press Release

Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues both Muscle and CNS Defects in Murine Pompe Disease

Researchers demonstrated that a newly developed AAV-PHP.B vector could robustly transduce both the central nervous system (CNS) and skeletal muscles in GAAKO mice. A single intravenous injection of an AAV-PHP.B vector expressing human GAA under the control of CMV enhancer/chicken β-actin promoter into two-week old GAAKO mice resulted in widespread GAA expression in the affected tissues. [Mol Ther Methods Clin Dev] Abstract | Full Article

AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome

To identify novel mechanistic pathways involved in Barth syndrome (BTHS) and evaluate the effects of gene therapy on proteomic profiles, the authors performed a multiplex tandem mass tagging (TMT) quantitative proteomics analysis to compare protein expression profiles from heart lysates isolated from BTHS, healthy wild-type, and BTHS treated with AAV9-TAZ gene replacement as neonates or adults. [Mol Ther Methods Clin Dev] Abstract | Graphical Abstract | Full Article

Preconditioned Adipose-Derived Stem Cells Ameliorate Cardiac Fibrosis by Regulating Macrophage Polarization in Infarcted Rat Hearts through the PI3K/STAT3 Pathway

Scientists investigated whether n-butylidenephthalide primed adipose-derived stem cells attenuated cardiac fibrosis via regulating macrophage phenotype by a PI3K/STAT3-dependent pathway in postinfarcted rats. [Lab Invest] Abstract

Safety and Efficacy Evaluations of an Adeno-Associated Virus Variant for Preparing IL10-Secreting Human Neural Stem Cell-Based Therapeutics

Interleukin-10 (IL10) secretion from human neural stem cells infected by rAAVr3.45 encoding IL10 did not alter the transcriptional profile of any gene by more than threefold, but the exogenously boosted IL10 was sufficient to provoke immunomodulatory effects in an ischemic brain injury animal model, thereby accelerating the recovery of neurological deficits and the reduction of brain infarction volume. [Gene Ther] Abstract

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The Journey to CAR T Cell Therapy: The Pediatric and Young Adult Experience with Relapsed or Refractory B-ALL

To improve outcomes of patients with relapsed or refractory disease, immunotherapies targeting specific B cell antigens are being developed. Tisagenlecleucel is an autologous anti-CD19 chimeric antigen receptor (CAR) T cell therapy recently approved by the FDA for patients with refractory leukemia or those with second or later relapse. [Blood Cancer J] Full Article

Manufacturing of Primed Mesenchymal Stromal Cells for Therapy

The authors examine how the choice of media, cell source, culture environment and storage affects the phenotype and clinical utility of mesenchymal stromal cell (MSC)-based products, and discuss the techniques better suited to prime MSCs with specific phenotypes of interest and the need for the continued development of standardized assays that provide quality assurance for clinical-grade MSCs. [Nat Biomed Eng] Abstract

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

Gamida Cell Announces Data from NAM-NK and NiCord^® Programs to Be Presented

Gamida Cell Ltd. announced that new data from its clinical programs will be presented. [Press release from Gamida Cell Ltd. discussing research to be presented at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR), Houston] Press Release

STEMCELL Technologies Launches mTeSR™ Plus, a Next-Generation Culture System for Human ES and iPS Cell Maintenance

STEMCELL Technologies has released mTeSR™ Plus, an enhanced version of mTeSR™1, the most widely published feeder-free human pluripotent stem cell maintenance medium. The new medium has undergone successful beta testing with several members of the Stem Cell COREdinates group and has completed rigorous in-house validation testing. [STEMCELL Technologies, Inc.] Press Release

Gamida Cell and Be The Match BioTherapies^® Announce Strategic Collaboration

Gamida Cell Ltd. and Be The Match BioTherapies^® announced a strategic collaboration to improve outcomes for patients undergoing allogeneic hematopoietic stem cell transplantation. [Gamida Cell Ltd.] Press Release

CRISPR Therapeutics and ProBioGen Sign Collaboration and License Agreement to Develop Novel In Vivo Delivery Technologies

CRISPR Therapeutics and ProBioGen announced a multi-year research collaboration focused on the development of novel in vivo delivery modalities for CRISPR/Cas9 leveraging ProBioGen’s existing technology and expertise. [CRISPR Therapeutics] Press Release

Alnylam Pharmaceuticals and Medison Pharma Partner to Commercialize RNAi Therapeutics in Israel

Alnylam Pharmaceuticals, Inc. and Medison Pharma announced an exclusive agreement to commercialize ONPATTRO^®, the first-ever commercialized RNAi therapeutic, as well as other investigational therapeutics under development in the Alnylam RNAi portfolio. [Alnylam Pharmaceuticals, Inc.] Press Release

Agenus Closes $150 Million Immuno-Oncology Transaction with Gilead

Agenus Inc. announced the closing of its immuno-oncology partnership deal with Gilead Sciences, Inc. focused on the development and commercialization of up to five novel immuno-oncology therapies. [Agenus Inc.] Press Release

Asterias Provides Top Line 12 Month Data Update for its OPC1 Phase I/IIa Clinical Trial in Severe Spinal Cord Injury

Asterias Biotherapeutics, Inc. provided top-line 12 month data from the company’s Phase I/IIa SCiStar study designed to evaluate the safety and potential efficacy of OPC1 in the treatment of severe cervical spinal cord injury. [Asterias Biotherapeutics, Inc.] Press Release

Athersys Announces Positive Results from Its Exploratory Clinical Study of MultiStem^® Cell Therapy for Treatment of Acute Respiratory Distress Syndrome (ARDS)

Athersys, Inc. announced summary results from its exploratory clinical study of the intravenous administration of MultiStem^® cell therapy to treat patients who are suffering from ARDS. [Athersys, Inc.] Press Release

Dicerna Announces Dosing of First Volunteer in Phase I Clinical Trial of DCR-HBVS Designed for the Treatment of Chronic Hepatitis B Virus

Dicerna Pharmaceuticals, Inc. announced the dosing of the first human volunteer in its Phase I clinical trial of DCR-HBVS, the company’s investigational GalXCTM-based therapy for the treatment of chronic hepatitis B virus infection in adults. [Dicerna Pharmaceuticals, Inc.] Press Release

Rocket Pharmaceuticals Announces Clearance of IND for RP-A501 Gene Therapy for Danon Disease

Rocket Pharmaceuticals, Inc. announced the clearance of the company’s Investigational New Drug (IND) application by the FDA for RP-A501. [Rocket Pharmaceuticals, Inc.] Press Release

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-130 in Huntington’s Disease

uniQure N.V. announced the FDA has completed its review of the company’s Investigational New Drug (IND) application for AMT-130, and the IND is now effective allowing uniQure to begin its planned Phase I/II study. [uniQure N.V.] Press Release

Allogene Therapeutics, in Collaboration with Servier, Announces FDA Clearance of the IND for ALLO-501, an anti-CD19 Allogeneic CAR T (AlloCAR T™) Therapy

Allogene Therapeutics, Inc. announced that the FDA has cleared Allogene’s Investigational New Drug (IND) application for ALLO-501 in patients with relapsed/refractory non-Hodgkin lymphoma. [Allogene Therapeutics, Inc.] Press Release

TC BioPharm Creates Allogeneic Cell Banks for CAR-T Cancer Therapy Products

TC BioPharm announced it has completed formulation of its first allogeneic GDT cell banks. The T cell banks provide TCB with core technology to develop a deep portfolio of next-generation CAR-T products directed against a wide variety of different cancer types, both hematological and solid tumors. [TC BioPharm Ltd.] Press Release

BrainStorm Granted Additional Patent for NurOwn^® in Israel

BrainStorm Cell Therapeutics Inc. announced that the Israel Patent Office has granted an additional patent titled “Methods of Generating Mesenchymal Stem Cells which Secrete Neurotrophic Factors.” [BrainStorm Cell Therapeutics Inc.] Press Release

Anixa Biosciences CAR-T Cancer Therapy Patent Receives Notice of Allowance

Anixa Biosciences, Inc. announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for the first patent covering Anixa subsidiary, Certainty Therapeutics, Inc.’s, CAR-T cancer treatment technology. [Anixa Biosciences, Inc.] Press Release

Talee Bio Announces $4.5 Million Award from the Cystic Fibrosis Foundation to Advance Development of Two Gene Therapy Product Candidates

Talee Bio, Inc. announced agreements with the Cystic Fibrosis Foundation to fund the preclinical development of two mutation-agnostic gene therapy product candidates, TL-101 and TL-102. [Talee Bio, Inc.] Press Release

GE Healthcare and G-CON to Offer Modular, Prefabricated Cell Therapy Manufacturing Lines and Cleanrooms

GE Healthcare has begun working with G-CON Manufacturing, makers of prefabricated cleanrooms, to offer a combined and deployable platform for early-stage manufacturing of cell therapies and viral vectors. [GE Healthcare (FierceBiotech)] Press Release

Orgenesis Announces U.S. Expansion of Masthercell Global Subsidiary

Orgenesis Inc. announced that its subsidiary, Masthercell Global Inc., is expanding into the United States and has executed a lease agreement to build a new 30,000 sq. ft manufacturing facility in Houston, Texas. [Orgenesis Inc.] Press Release

Lovelace Biomedical Wins 5 Year Gene Therapy Contract

Lovelace Biomedical has won a third consecutive five-year support contract to provide pharmacology and toxicology drug development services to the Gene Therapy Resource Program at the National Heart, Lung, and Blood Institute. [Lovelace Biomedical (PR Newswire Association LLC.)] Press Release

Japan’s Approval of Stem-Cell Treatment for Spinal-Cord Injury Concerns Scientists

Japan has approved a stem-cell treatment for spinal-cord injuries. The event marks the first such therapy for this kind of injury to receive government approval for sale to patients. [Nature News] Editorial

FDA Staff Return to Work, but Threat of Second Shutdown Looms

FDA staff are returning to paid work after the longest government shutdown in U.S. history. But with temporary funding set to lapse Feb. 15 and President Donald Trump skeptical a deal can be reached, the agency may be plunged back into a shutdown in less than three weeks. [FierceBiotech] Editorial

NIH

Prospective Grant of an Exclusive Patent License: “Multifunctional RNA Nanoparticles and Methods of Uses” and “RNA/DNA Hybrid Nanoparticles Modified with Single Stranded RNA Toeholds and Uses Thereof” (FR Doc. No:2018-27671) Notice

NEW Keystone Symposia: Protein Replacement through Nucleic Acid Therapies
April 7-10, 2019
Steamboat Springs, Colorado, United States

NEW 2019 Stanford Drug Discovery Symposium
April 22-23, 2019
Palo Alto, California, United States

NEW The NYSCF Conference 2019
October 22, 2019
New York, New York, United States

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NEW Research Associate – Bioengineering (STEMCELL Technologies Inc.)

NEW 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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