Cell Therapy News Volume 19.27 | Aug 13 2018

TOP STORY

Targeted Delivery of a PD-1-Blocking scFv by CAR-T Cells Enhances Anti-Tumor Efficacy In Vivo

Researchers modified CAR-T cells to secrete PD-1-blocking single-chain variable fragments (scFv). These scFv-secreting CAR-T cells acted in both a paracrine and autocrine manner to improve the anti-tumor activity of CAR-T cells and bystander tumor-specific T cells in clinically relevant syngeneic and xenogeneic mouse models of PD-L1⁺ hematologic and solid tumors. [Nat Biotechnol] Abstract | Press Release

(Ranked by impact factor of the journal)

Characterization and Transplantation of CD73-Positive Photoreceptors Isolated from Human iPSC-Derived Retinal Organoids

Scientists demonstrated that CD73 targeting by magnetic-activated cell sorting is an effective strategy to separate a safe population of transplantable photoreceptors. CD73⁺ photoreceptor precursors could be isolated in large numbers and transplanted into rat eyes, showing capacity to survive and mature in close proximity to host inner retina of a model of photoreceptor degeneration. [Stem Cell Reports] Full Article | Graphical Abstract

Prodrug Suicide Gene Therapy for Cancer Targeted Intracellular by Mesenchymal Stem Cell Exosomes

When conditional medium was applied to tumor cells, exosomes were internalized by recipient tumor cells and in the presence of the prodrug 5-fluorocytosine (5-FC) effectively triggered dose-dependent tumor cell death by endocytosed exosomes via an intracellular conversion of the prodrug 5-FC to 5-fluorouracil. [Int J Cancer] Abstract

Antitumor Virotherapy Using Syngeneic or Allogeneic Mesenchymal Stem Cell Carriers Induces Systemic Immune Response and Intratumoral Leukocyte Infiltration in Mice

The authors used Celyvir with syngeneic and allogeneic mouse mesenchymal stem cells to determine their antitumor efficacy in a C57BL/6 murine adenocarcinoma model. [Cancer Immunol Immunother] Abstract

Development of an Inducible Anti-VEGF rAAV Gene Therapy Strategy for the Treatment of Wet AMD

Scientists modulated the intraocular concentration of Eylea following recombinant adeno-associated virus (rAAV) delivery, leading to nearly complete inhibition of clinically significant choroidal neovascularization lesions in an established mouse model of wet age-related macular degeneration (AMD). [Sci Rep] Full Article

Combinatorial Prg4 and Il-1ra Gene Therapy Protects against Hyperalgesia and Cartilage Degeneration in Post-Traumatic Osteoarthritis

Employing two surgical techniques to model moderate and severe post-traumatic osteoarthritis, scientists found that combined delivery of helper-dependent adenoviruses expressing interleukin-1 receptor antagonist (IL-1Ra) and lubricin (PRG4) preserved articular cartilage better than either monotherapy in both models as demonstrated by preservation of articular cartilage volume and surface area. [Hum Gene Ther] Abstract

Ectopic BAT mUCP-1 Overexpression in SKM by Delivering a BMP7/PRDM16/PGC-1a Gene Cocktail or Single PRMD16 Using Non-Viral UTMD Gene Therapy

Researchers used ultrasound-targeted microbubble destruction (UTMD), a novel targeted, non-viral approach to gene therapy, to deliver genes in the brown adipose tissue (BAT) differentiation pathway into rodent skeletal muscle (SKM) to engineer a thermogenic BAT phenotype with ectopic mUCP-1 overexpression. [Gene Ther] Abstract

Alveolar Bone Regeneration during Three Weeks following Transfer of BMP-2/7 Gene via In Vivo Electroporation

Investigators analyzed alveolar bone during a period of three weeks following gene transfer to periodontal tissue. Non-viral plasmid vector pCAGGS-BMP-2/7 or pCAGGS control was injected into palatal periodontal tissue of the first molar of the rat maxilla and immediately electroporated with 32 pulses of 50 V for 50 msec. [Eur J Histochem] Abstract | Full Article

Human Adipose-Derived CD146⁺ Stem Cells Increase Life Span of a Muscular Dystrophy Mouse Model More Efficiently than Mesenchymal Stromal Cells

The authors analyzed the effect of CD146⁺ cells compared to mesenchymal stem/stromal cells derived from the same human adipose sample when injected in the dko mouse model without immunosuppression. [DNA Cell Biol] Abstract

Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News.

Gene Therapy for Neurological Disorders: Progress and Prospects

Scientists discuss key considerations and challenges in the future design and development of therapeutic adeno-associated viral vectors, highlighting the most promising targets and recent clinical advances. [Nat Rev Drug Discov] Abstract

High-Dose Melphalan and Stem Cell Transplantation in Systemic AL Amyloidosis in the Era of Novel Anti-Plasma Cell Therapy: A Comprehensive Review

The authors touch on the history of stem cell transplant (SCT) for amyloidosis (AL) and examine the data on eligibility, mobilization, induction, risk-adapted melphalan dosing, engraftment, consolidation and maintenance, and long-term outcomes with SCT. [Bone Marrow Transplant] Abstract

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

Mustang Bio and St. Jude Children’s Research Hospital Enter into Exclusive Worldwide License Agreement for a Ground-Breaking, Clinical-Stage Lentiviral Gene Therapy with Curative Potential for X-Linked Severe Combined Immunodeficiency

Mustang Bio, Inc. and St. Jude Children’s Research Hospital announced that they have partnered and entered into an exclusive worldwide license agreement for the development of a first-in-class ex vivo lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease. [Mustang Bio, Inc.] Press Release

Fortress Biotech Announces Aevitas Therapeutics Enters Sponsored Research Agreement with the University of Pennsylvania to Advance AAV Gene Therapy Technology

Fortress Biotech announced that its subsidiary, Aevitas Therapeutics, Inc., has entered into a sponsored research agreement with the laboratory of Wenchao Song, Ph.D., a professor of Pharmacology in the Perelman School of Medicine at the University of Pennsylvania, to evaluate Aevitas’ adeno-associated virus gene therapy technology in Dr. Song’s animal models of complement-mediated diseases. [Fortress Biotech] Press Release

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, Imperial Innovations and Oxford BioMedica Announce New Partnership to Develop First-in-Class Gene Therapy for Cystic Fibrosis

Boehringer Ingelheim and Oxford BioMedica announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis. [Boehringer Ingelheim International GmbH] Press Release

Regeneron and bluebird bio Announce Collaboration to Discover, Develop and Commercialize New Cell Therapies for Cancer

Regeneron Pharmaceuticals, Inc. and bluebird bio, Inc. announced a collaboration to apply their respective technology platforms to the discovery, development and commercialization of novel immune cell therapies for cancer. [Regeneron Pharmaceuticals, Inc.] Press Release

Avalon GloboCare Forms Strategic Partnership with Weill Cornell Medical College to Co-Develop Technologies and Bio-Production of CAR-T Therapy

Avalon GloboCare Corp. announced that the company has formed a strategic partnership with Weill Cornell’s cGMP Cellular Therapy Facility and Laboratory for Advanced Cellular Engineering headed by Dr. Yen-Michael Hsu. [Avalon GloboCare Corp.] Press Release

Allergan and Editas Medicine Announce Exercise of Options to Jointly Develop CRISPR Genome Editing Experimental Medicine EDIT-101

Allergan plc and Editas Medicine, Inc. announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited, has exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. [Allergan plc] Press Release

Alnylam Announces First-Ever FDA Approval of an RNAi Therapeutic, ONPATTRO™ (Patisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Pharmaceuticals, Inc. announced that the FDA approved ONPATTRO™ lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. [Alnylam Pharmaceuticals, Inc.] Press Release

RXi Pharmaceuticals Announces Positive Results from Phase I/II Trial with RXi-109 for Retinal Scarring

RXi Pharmaceuticals Corporation announced positive results with RXI-109 in a Phase I/II clinical trial. [RXi Pharmaceuticals Corporation] Press Release

ProtoKinetix Enters into 3^rd Phase of Retinal Cell Replacement Therapy Testing at UBC

ProtoKinetix, Incorporated has entered into the 3^rd phase of testing in retinal cell replacement therapy at the University of British Columbia (UBC). [ProtoKinetix, Incorporated] Press Release

Sangamo Announces Positive Preliminary Data from the Phase I/II Alta Study Evaluating SB-525 Gene Therapy for Hemophilia A

Sangamo Therapeutics, Inc. announced positive preliminary data from the Phase I/II clinical trial evaluating SB-525, a cDNA gene therapy candidate for hemophilia A. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer Inc. for the development and commercialization of potential gene therapy programs for hemophilia A. [Sangamo Therapeutics, Inc.] Press Release

Unum Therapeutics Announces Active Investigational New Drug (IND) Application for Antibody-Coupled T Cell Receptor (ACTR) platform in Combination with Trastuzumab in Patients with HER2⁺ Advanced Cancers

Unum Therapeutics Inc. announced that an IND application is now active for ACTR T cells in combination with trastuzumab for the treatment of patients with HER2⁺ advanced cancers. [Unum Therapeutics Inc.] Press Release

US Congress Leaves Science Agencies Hanging — Again

Lawmakers in the US Congress are running out of time to pass a budget for the 2019 fiscal year, and have yet to resolve major disagreements over climate-change and environment programs. [Nature News] Editorial

Gottlieb: FDA Will Streamline Drug Safety Evaluations

The FDA will soon standardize the way it handles data on the safety and effectiveness of drugs in an effort to reduce inconsistencies in the drug review process. [STAT News] Editorial

India Cracks Down on Plagiarism at Universities

India has for the first time introduced regulations to detect and punish acts of plagiarism at universities. Punishments for researchers or students caught breaking the rules range from requiring that a manuscript be withdrawn to sacking or expulsion, depending on the extent of the plagiarism. [Nature News] Editorial

Former Students Allege Mistreatment of Lab Animals at Calgary

An investigation is underway into the treatment of lab animals at the University of Calgary after several former students complained, CBC reported. Allegations center on the now-closed lab of neuropsychologist Vedran Lovic, and include reports of rodents regularly waking up mid-surgery in pain due to a lack of anesthetic. [The Scientist] Editorial

FDA

Revocation of Authorization of Emergency Use of an In Vitro Diagnostic Device for Detection of Ebola Virus (FR Doc. No:2018-16537) Notice

NEW Cell Therapies and Bioengineering Conference
September 20-22, 2018
San Francisco, California, United States

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

NEW Molecular Biologist (STEMCELL Technologies Inc.)

NEW Postdoctoral Position – Gene Therapy (Genethon)

Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research)

Postdoctoral Scientist – Novel T Cell Based Cancer Therapies (Netherlands Cancer Institute – Antoni van Leeuwenhoek)

Research Associate – Neuroimmunology (Thomas Jefferson University)

Research Assistant or Associate – Cell Therapy (California Institute for Biomedical Research)

Postdoctoral Fellow – DNA Damage Repair and Pediatric Sarcoma Therapy (University of Texas Health Science Center at San Antonio)

Postdoctoral Position – Corneal Diseases (University of California, Los Angeles)

PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

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

Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or suggestions? Submit your feedback here.