Cell Therapy News Volume 19.29 | Aug 27 2018

TOP STORY

Deciduous Autologous Tooth Stem Cells Regenerate Dental Pulp after Implantation into Injured Teeth

The authors showed that implantation of autologous tooth stem cells from deciduous teeth regenerated dental pulp with an odontoblast layer, blood vessels, and nerves in two animal models. [Sci Transl Med] Abstract

(Ranked by impact factor of the journal)

Mutation-Independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV Vector

Scientists identified a highly efficient shRNA that targets human (and canine) rhodopsin (RHO) in a mutation-independent manner. In a single adeno-associated viral (AAV) vector they combined this shRNA with a human RHO replacement cDNA made resistant to RNA interference and tested this construct in a naturally occurring canine model of RHO-autosomal dominant retinitis pigmentosa. [Proc Natl Acad Sci USA] Abstract | Press Release

Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma

Researchers combined adeno-associated viral retinal gene therapy with the targeted C3 inhibitor CR2-Crry. They showed that intravitreal injection of AAV2.CR2-Crry produced sustained Crry overexpression in the retina, and reduced deposition of the activation product complement C3d on retinal ganglion cells and the inner retina of DBA/2J mice. [Mol Ther] Abstract

AAV9-Mediated Telomerase Activation Does Not Accelerate Tumorigenesis in the Context of Oncogenic K-Ras-Induced Lung Cancer

Investigators tested the safety of adeno-associated viral vector (AAV)9-mediated telomerase (Tert) overexpression in the context of a cancer prone mouse model, owing to expression of oncogenic K-ras. As control, they also treated mice with AAV9 vectors carrying a catalytically inactive form of Tert, known to inhibit endogenous telomerase activity. [PLoS Genet] Full Article | Press Release

Y-27632 Preconditioning Enhances Transplantation of Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes in Myocardial Infarction Mice

Researchers investigated whether preconditioning of cardiomyocytes, derived from human induced-pluripotent stem cells, with Y-27632 improves their survival and engraftment in a murine model of acute myocardial infarction. [Cardiovasc Res] Abstract

An Allogenic Therapeutic Strategy for Canine Spinal Cord Injury Using Mesenchymal Stem Cells

Successfully transfected cells were injected around the skin wound in mice and in vivo imaging was done at 6, 12 and 24 hours post mesenchymal stem cell (MSC) delivery. In vivo imaging revealed that transfected bone marrow-derived MSCs migrated and concentrated predominantly toward the center of the wound. [J Cell Physiol] Abstract

In Vivo Distribution of U87MG Cells Injected into the Lateral Ventricle of Rats with Spinal Cord Injury

Scientists analyzed in vivo distribution of cells that were injected into lateral ventricles of rats with spinal cord injury at thoracic level using in vivo imaging techniques. [PLoS One] Full Article

Adenoviral Delivery of VHL Suppresses Bone Sarcoma Cell Growth through Inhibition of Wnt/β-Catenin Signaling

Researchers constructed a recombinant adenovirus 5-VHL vector and evaluated its expression in bone sarcomas, and antitumor activity in vitro and in vivo. [Cancer Gene Ther] Abstract

Disc Cell Therapy with Bone-Marrow-Derived Autologous Mesenchymal Stromal Cells in a Large Porcine Disc Degeneration Model

Fibrin matrix-assisted autologous bone-marrow-derived mesenchymal stromal cell therapy was compared to acellular fibrin matrix therapy in a porcine in vivo model. [Eur Spine J] Abstract

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

Cell Therapy in Idiopathic Pulmonary Fibrosis

Idiopathic pulmonary fibrosis is a fatal disease with no effective or curative treatment options. In recent decades, cell-based therapies using stem cells or lung progenitor cells to regenerate lung tissue have experienced rapid growth in both preclinical animal models and translational clinical studies. [Med Sci (Basel)] Full Article

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

Applied DNA Presenting Linear DNA for Gene Therapy

Applied DNA Sciences Inc. will present a poster entitled “Linear DNA High-Expression Amplicons for Gene Therapy”. [Press release from Applied DNA Sciences Inc. discussing research to be presented at the 6^th Annual Immuno-Oncology Summit, Boston] Press Release

bluebird bio and Gritstone Oncology Announce Strategic Collaboration to Develop Novel Cancer Cell Therapies

bluebird bio, Inc. and Gritstone Oncology, Inc. announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy. [bluebird bio, Inc.] Press Release

Orgenesis Announces Collaboration with Ben-Gurion University to Develop and Commercialize a Novel Scaffold Technology for Cell Transplantation

Orgenesis Inc. along with BGN Technologies and the National Institute for Biotechnology in the Negev announced a collaboration to develop and commercialize a novel alginate scaffold technology for cell transplantation, with an initial focus on autoimmune diseases. [Orgenesis Inc.] Press Release

Alnylam Completes Enrollment in ENVISION Phase III Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias (AHPs)

Alnylam Pharmaceuticals, Inc. announced that it has achieved full patient accrual in its ENVISION Phase III study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of AHPs. [Alnylam Pharmaceuticals, Inc.] Press Release

BrainStorm Cell Therapeutics Announces Successful Interim Safety Analysis in Phase III Trial of NurOwn^® Therapy for ALS

BrainStorm Cell Therapeutics Inc. announced that the independent Data Safety Monitoring Board has completed the pre-specified interim analysis of safety outcomes for the first 31 participants treated with NurOwn^® in the Phase III trial in ALS. [BrainStorm Cell Therapeutics Inc.] Press Release

Audentes Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by the FDA to AT132 for the Treatment of X-Linked Myotubular Myopathy

Audentes Therapeutics, Inc. announced that the U.S. FDA granted RMAT designation to AT132 for the treatment of X-linked myotubular myopathy (XLMTM). RMAT designation for AT132 was granted based on positive interim clinical data from ASPIRO, the ongoing Phase I/II clinical study of AT132 for XLMTM. [Audentes Therapeutics, Inc.] Press Release

Novartis Receives European Commission Approval of Its CAR-T Cell Therapy, Kymriah^® (Tisagenlecleucel)

Novartis announced that the European Commission has approved Kymriah^®. The approved indications are for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. [Novartis AG] Press Release

uniQure Announces First Patient Treated in Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B

uniQure N.V. announced that it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release

MeiraGTx Announces AAV-CNGA3 Granted Rare Pediatric Disease Designation by the U.S. FDA for the Treatment of Achromatopsia

MeiraGTx Holdings Plc announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation to the company’s gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia due to mutations in the CNGA3 gene. [MeiraGTx Holdings Plc] Press Release

Adverum Biotechnologies Receives Orphan Drug Designation for ADVM-053, a Novel Gene Therapy Candidate for the Treatment of Hereditary Angioedema

Adverum Biotechnologies, Inc. announced that the FDA has granted Orphan Drug Designation to ADVM-053, a preclinical gene therapy candidate being investigated as a potential single-administration treatment which has the potential to provide sustained levels of the C1 esterase inhibitor protein. [Adverum Biotechnologies, Inc.] Press Release

No-Deal Brexit ‘Worse than Thought’ for Science

The impact of a no-deal Brexit on British science could be worse than previously thought, according to a new analysis. The campaign group Scientists for EU has studied the Brexit technical notes released by the government. One of the documents states the UK would no longer be eligible for three of the EU’s major funding programs. [The BBC] Editorial

Senate Approves $2B Boost for NIH in $850B+ Spending Bill

For the fourth year in a row, the Senate has approved a significant increase in funding for the National Institutes of Health (NIH). The $2 billion boost raises the agency’s funding about 5% to $39.1 billion for fiscal year 2019 and comes as part of an $854 billion package combining spending bills for the Departments of Defense, Health and Human Services, Education and Labor. [FierceBiotech] Editorial

AAAS Insiders Petition to Revoke Honors of Sexual Harassers

Using the hashtag #TimesUpAAAS, 36 researchers recognized by the American Association for the Advancement of Science (AAAS) for their science communication work posted an open letter asking the society not to bestow honors on those “who have engaged in harassment and discrimination.” The organization says it’s working on a policy. [The Scientist] Editorial

To Highlight Gender Gaps, Scientists Decline Opportunities

Activists are protesting imbalanced conferences, editorial boards, and other professional activities by refusing to join. When neuroscientist Rogier Kievit was invited to join a journal’s editorial board, he took a look at the skewed gender ratio of its current members—21 men and 3 women—and said no thanks. [The Scientist] Editorial

Dartmouth Misconduct Case Highlights the Mistreatment of Junior Scientists

The Dartmouth Institute for Health Policy and Clinical Practice probably isn’t the most collegial of places these days. That’s because Dartmouth officials agreed with Samir Soneji, an associate professor at the New Hampshire University, that his colleague Dr. H. Gilbert Welch, a leading expert on cancer screening and overtreatment, used data that belonged to Soneji and a collaborator in California in a subsequent publication without even a hat tip. [STAT News] Editorial

NEW Alliance for Regenerative Medicine: Cell & Gene Meeting on the Mesa 2018
October 3-5, 2018
La Jolla, California, United States

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

NEW Senior Scientist – CAR-T Cell Research (CELLECTIS Inc.)

Molecular Biologist (STEMCELL Technologies Inc.)

Postdoctoral Fellow – Immuno-Oncology (CELLECTIS Inc.)

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)

PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

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

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

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