Cell Therapy News Volume 20.07 | Mar 4 2019

TOP STORY

An Automated Hybrid Bioelectronic System for Autogenous Restoration of Sinus Rhythm in Atrial Fibrillation

Scientists present an automated hybrid bioelectronic system for shock-free termination of atrial fibrillation that enabled the heart to act as an electric current generator for autogenous restoration of sinus rhythm. They showed that local, right atrial delivery of adenoassociated virus vectors encoding a light-gated depolarizing ion channel resulted in efficient and spatially confined transgene expression. [Sci Transl Med] Abstract

(Ranked by impact factor of the journal)

Reactive Cholangiocytes Differentiate into Proliferative Hepatocytes with Efficient DNA Repair in Mice with Chronic Liver Injury

Researchers tracked cholangiocytes using osteopontin-iCreER^T2 and hepatocytes with AAV8-TBG-Cre. Mice received carbon tetrachloride for >24weeks to induce chronic liver injury. Livers were collected for reporter proteins, cell proliferation and death, DNA damage, and nuclear ploidy analysis; hepatocytes were isolated for RNAseq. [J Hepatol] Full Article | Graphical Abstract

Efficacy of Systemic Temozolomide‐Activated Phage‐Targeted Gene Therapy in Human Glioblastoma

The authors investigated systemic combination therapy using temozolomide (TMZ) and targeted suicide gene therapy by the RGD4C/AAVP‐Grp78. In vitro they showed that TMZ increased endogenous Grp78 gene expression and boosted transgene expression from the RGD4C/AAVP‐Grp78 in human glioblastoma multiforme cells. Next, RGD4C/AAVP‐Grp78 targeted intracranial tumors in mice following intravenous administration. [EMBO Mol Med] Full Article | Graphical Abstract

Constitutively Active MyD88/CD40 Costimulation Enhances Expansion and Efficacy of Chimeric Antigen Receptor T Cells Targeting Hematological Malignancies

Scientists describe a novel CAR-T cell platform that utilized expression of the toll-like receptor adaptor molecule, MyD88, and tumor-necrosis factor family member, CD40, tethered to the CAR molecule through an intentionally inefficient 2A linker system, providing a constitutive signal that drove CAR-T survival, proliferation, and antitumor activity against CD19⁺ and CD123⁺ hematological cancers. [Leukemia] Full Article

Hypoxia Mimicking Hydrogels to Regulate the Fate of Transplanted Stem Cells

In vivo, dimethyloxalylglycine delivery significantly reduced mineralization of the proteoglycan-rich cartilaginous tissue generated by mesenchymal stem cells within alginate hydrogels loaded with TGF-β3 and BMP-2. [Acta Biomater] Abstract | Graphical Abstract

Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease

Investigators evaluated the selective ββ2 agonist salmeterol in α-glucosidase (GAA) knockout mice in combination with an adeno-associated virus vector expressing human GAA specifically in the liver. [Hum Gene Ther] Abstract

Nasal Delivery of Fasudil‐Modified Immune Cells Exhibits Therapeutic Potential in Experimental Autoimmune Encephalomyelitis

Experimental autoimmune encephalomyelitis was induced with myelin oligodendrocyte glycoprotein 35‐55 in C57BL/6 mice, and encephalitogenic mononuclear cells were treated with Fasudil in vitro. Mice received 3×10⁶ cells/10 μL per nasal cavity on day 3 and 11 postimmunization, respectively. [CNS Neurosci Ther] Full Article

Mesenchymal Stem Cell Therapy in Submandibular Salivary Gland Allotransplantation

Investigators evaluated the effect of allogeneic mesenchymal stem cell therapy on the survival of allotransplanted submandibular salivary glands. [Transplantation] Abstract

Salmonella-Mediated Therapy Targeting Indoleamine 2, 3-Dioxygenase 1 (IDO) Activates Innate Immunity and Mitigates Colorectal Cancer Growth

In vivo, S. typhimurium delivering an shRNA plasmid targeting IDO treatment significantly delayed CT26 and MC38 tumor progression compared to mice treated with scrambled shRNA control or the clinically tested IDO inhibitor epacadostat. [Cancer Gene Ther] Full Article

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

Gene Therapy in Rheumatoid Arthritis: Strategies to Select Therapeutic Genes

Important strategies used to select effective genes for rheumatoid arthritis gene therapy are outlined. [J Cell Physiol] Abstract

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

Atara Biotherapeutics Announces Presentations Highlighting Next-Generation CAR T Platform and Mesothelin-Targeted CAR T Clinical Results

Atara Biotherapeutics, Inc. announced presentations highlighting next-generation CAR T platform and mesothelin-targeted CAR T clinical safety and efficacy results from Memorial Sloan Kettering Cancer Center. [Press release from Atara Biotherapeutics, Inc. discussing research to be presented at the American Association of Cancer Research (AACR) Annual Meeting 2019, Atlanta] Press Release

Sensei Biotherapeutics to Present First Preclinical Data on SNS-723 and Long-Term Clinical Results on SNS-301 Data

Sensei Biotherapeutics, Inc. announced that it will present novel preclinical data on SNS-723, a first-in-class CAR-T cell therapy and additional Phase I data on the long-term effects of SNS-301, a first-in-class cancer immunotherapy, both targeting a novel tumor specific embryonic antigen, human aspartate β-hydroxylase. [Press release from Sensei Biotherapeutics, Inc. discussing research to be presented at the American Association of Cancer Research (AACR) Annual Meeting 2019, Atlanta] Press Release

uniQure Presents New Preclinical Data on AMT-130

uniQure N.V. presented new preclinical data on AMT-130, its gene therapy candidate for the treatment of Huntington’s disease. [Press release from uniQure N.V. discussing research presented at the 14^th Annual CHDI Huntington’s Disease Therapeutics Conference, Palm Springs] Press Release

MaxCyte Announces Multi-Drug Clinical & Commercial Agreement with Kite, a Gilead Company

MaxCyte announced that it has expanded its relationship with Kite, a Gilead Company, by entering into a multi-drug clinical and commercial agreement. [MaxCyte, Inc.] Press Release

Biogen Announces Agreement to Acquire Nightstar Therapeutics to Establish Clinical Pipeline of Gene Therapy Candidates in Ophthalmology

Biogen announced that it has entered into an agreement to acquire Nightstar Therapeutics. [Biogen] Press Release

NantKwest Signs Worldwide License Agreement with ProMab Biotechnologies to Exclusively Develop Select Next Generation Targeted Natural Killer Cell Therapeutics

NantKwest, Inc. and ProMab Biotechnologies announced the establishment of a worldwide license to a B-Cell Maturation Antigen targeted antibody sequence for multiple myeloma along with an option for up to five undisclosed targeting sequences for exclusive use in the development of chimeric antigen receptor based natural killer cell therapies. [NantKwest, Inc.] Press Release

Kadimastem Announces the Completion of Cell Transplantation of AstroRx^® in ALS Patients for Cohort A

Kadimastem announced the completion of cell transplantation (AstroRx^®) in five patients who were enrolled in cohort A for its clinical trial in ALS. The Phase I/IIa clinical trial is being conducted by the Department of Neurology of the Hadassah Ein-Kerem Medical Center, and it is expected to include a total of 21 patients. [Kadimastem] Press Release

Sarepta Therapeutics Announces Positive and Robust Expression and Biomarker Data from the First Three-Patient Cohort Dosed in the MYO-101 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy

Sarepta Therapeutics, Inc. announced positive results from three Limb-girdle muscular dystrophy (LGMD) Type 2E clinical trial participants who received MYO-101. MYO-101 is a novel gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-SG protein, the lack of which causes LGMD2E. [Sarepta Therapeutics, Inc.] Press Release

Hemostemix Announces UC Davis Health Added as a Trial Site for Phase II Clinical Trial

Hemostemix Inc. announced that it has received all approvals the UC Davis Health located Sacramento, California to be clinical trial site for the company’s Phase II clinical trial for critical limb ischemia. [Hemostemix Inc.] Press Release

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

CARsgen Therapeutics announced that one of its leading drug candidates, CT053 fully human BCMA (B-Cell Maturation Antigen)-CAR-T cell for the treatment of patients suffering from relapsed/refractory multiple myeloma, has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA). [CARsgen Therapeutics (PR Newswire Association LLC.)] Press Release

Avalon GloboCare Announces Filing of Provisional Patent Applications for AVA-101, a Novel Transposon-Based, Multi-Targeted CAR-T Therapy

Avalon GloboCare Corp. announced that the company and Arbele Limited have jointly filed provisional patent applications covering method and composition of matter claims for AVA-101, a novel multi-targeted, transposon-based chimeric antigen receptor (CAR)-T cellular immunotherapy. [Avalon GloboCare Corp.] Press Release

Cell Medica Awarded USD 8.7 Million CPRIT Grant to Accelerate CMD-502 Off-the-Shelf CAR-NKT Cell Therapy into Clinical Development

Cell Medica announced that it has been awarded an $8.7 million research grant from the Cancer Prevention and Research Institute of Texas (CPRIT). The grant will support preclinical and clinical development of the company’s off-the-shelf chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies to treat hematological and solid tumors. [Cell Medica] Press Release

The CRISPR-baby Scandal: What’s Next for Human Gene-Editing

In the three months since He Jiankui announced the birth of twin girls with edited genomes, the questions facing the scientific community have grown knottier. [Nature News] Editorial

China Unveils New Rules on Biotech after Gene-Editing Scandal

China has unveiled draft regulations on gene editing and other potentially risky biomedical technologies after a Chinese scientist’s claim of helping to create gene-edited babies roiled the global science community. [STAT News] Editorial

Peer-Review Experiments Tracked in Online Repository

A grass-roots group of biologists has started a website to keep track of the proliferation of experiments in academic peer review — including trials and platforms aimed at making the review process faster, cheaper, and more transparent and interactive. [Nature News] Editorial

NIH Letters Asking about Undisclosed Foreign Ties Rattle U.S. Universities

The National Institutes of Health (NIH) has recently sent letters to dozens of major U.S. research universities asking them to provide information about specific faculty members with NIH funding who are believed to have links to foreign governments that the Bethesda, Maryland–based institute did not know about. [ScienceInsider] Editorial

NEW World Advanced Therapies and Regenerative Medicine Congress 2019
May 15-17, 2019
London, United Kingdom

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

NEW Associate Scientist – Immuno-Oncology (HMH Center for Discovery & Innovation)

NEW Stem Cell Podcast Co-Host (Stem Cell Podcast)

Research Technologist – Immunology (STEMCELL Technologies Inc.)

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

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

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)

Cell Manufacturing Scientist (CSL Behring)

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

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

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

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

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.