Cell Therapy News Volume 20.11 | Apr 1 2019

TOP STORY

Intramyocardial Injection of Mesenchymal Precursor Cells and Successful Temporary Weaning from Left Ventricular Assist Device Support in Patients with Advanced Heart Failure

Among patients with advanced heart failure, intramyocardial injections of mesenchymal precursor cells, compared with injections of a cryoprotective medium as sham treatment, did not improve successful temporary weaning from left ventricular assist device support at six months. [JAMA] Abstract

(Ranked by impact factor of the journal)

Photothermal Therapy Promotes Tumor Infiltration and Antitumor Activity of CAR T Cells

Investigators found that chondroitin sulfate proteoglycan‐4‐specific CAR T cells infused in Nod scid gamma mice engrafted with the human melanoma WM115 cell line had superior antitumor activity after photothermal ablation of the tumor. [Adv Mater] Abstract | Press Release

Bioengineered Human Acellular Vessels Recellularize and Evolve into Living Blood Vessels after Human Implantation

The authors developed an investigational bioengineered human acellular vessel, which is currently being studied as a hemodialysis conduit in patients with end-stage renal disease. [Sci Transl Med] Abstract | Press Release | Editorial

Gene Replacement Therapy in a Model of Charcot-Marie-Tooth 4C Neuropathy

Scientists developed a gene replacement therapy for treating Charcot-Marie-Tooth disease type 4C to rescue the phenotype of the Sh3tc2^−/− mouse model. They generated a lentiviral vector LV-Mpz.SH3TC2.myc to drive expression of the human SH3TC2 cDNA under the control of the Mpz promoter specifically in myelinating Schwann cells. [Brain] Full Article

CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters

Researchers describe a novel approach based on CRISPR/dCas9 transcriptional activation to achieve robust transgene expression from transgene cassettes containing tissue or cell type-specific promoters after infection with AAV vectors in cell-based systems. [Mol Ther Methods Clin Dev] Abstract | Full Article

Early Intravenous Infusion of Mesenchymal Stromal Cells Exerts a Tissue Source Age‐Dependent Beneficial Effect on Neurovascular Integrity and Neurobehavioral Recovery after Traumatic Cervical Spinal Cord Injury

A moderately severe cervical clip compression/contusion injury was induced at C7‐T1 in adult female rats, followed by an intravenous tail vein infusion one‐hour post‐spinal cord injury of term‐birth human umbilical cord perivascular cells, first‐trimester human umbilical cord perivascular cells, adult bone marrow mesenchymal stem cells, or vehicle control. [Stem Cells Transl Med] Full Article

Combination of Cabazitaxel and p53 Gene Therapy Abolishes Prostate Carcinoma Tumor Growth

In a xenograft mouse model of in situ gene therapy, AdRGD-PGp53 treatment, when combined with cabazitaxel, drastically reduced tumor progression and increased survival rates to 100%. [Gene Ther] Abstract

Safe and Neuroprotective Vectors for Long-Term Traumatic Brain Injury Gene Therapy

The authors evaluated in the controlled cortical impact model of traumatic brain injury, the safety of integrative-deficient lentiviral vectors or the non-viral HNRK recombinant modular protein/DNA nanovector. [Gene Ther] Abstract

Safety and Efficacy of Oncolytic HSV-1 G207 Inoculated into the Cerebellum of Mice

Scientists demonstrated that cerebellar inoculation of G207 was systemically non-toxic in an immunocompetent, herpes simplex virus (HSV)-1 sensitive mouse strain. Mice had neither abnormal brain/organ pathology nor evidence of G207 replication by immunohistochemistry at days 7 and 30 after cerebellar G207 inoculation. [Cancer Gene Ther] Abstract

Combination of Stem Cell and Gene Therapy Ameliorates Symptoms in Huntington’s Disease Mice

Researchers demonstrated the efficacy of the combination of stem cell and gene therapy in a transgenic Huntington’s disease mouse model using rhesus monkey neural progenitor cells. [npj Regen Med] Full Article

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CAR T Cell Toxicity – Current Management and Future Directions

The authors offer a practical guide for the recognition and management of the most important toxicities related to the use of current commercial CAR T cells, and also highlight strategies to diminish these side effects in the future. [HemaSphere] Abstract

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

Orchard Therapeutics Presents New Registrational Data Demonstrating Sustained Clinical Benefit of OTL-200 for the Treatment of MLD

Orchard Therapeutics presented the full registrational dataset from a 20 patient trial evaluating the efficacy and safety of OTL-200, an ex vivo autologous hematopoietic stem cell-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). [Press release from Orchard Therapeutics discussing research presented at the 45^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Frankfurt] Press Release

Two BrainStorm Scientific Abstracts Accepted for Presentation

BrainStorm Cell Therapeutics Inc. announced that two scientific abstracts have been accepted for presentation. [Press release from BrainStorm Cell Therapeutics Inc. discussing research presented at the 71^st American Academy of Neurology (AAN) Annual Meeting, Philadelphia] Press Release

Dicerna™ Announces the Presentation of Updated Data Demonstrating Utility of Its Lead Compound DCR-PHXC in Treating Primary Hyperoxaluria Type 1 (PH1) and Type 2 (PH2)

Dicerna™ Pharmaceuticals, Inc. announced the presentation of updated data from its ongoing PHYOX™1 Phase I clinical trial evaluating DCR-PHXC, the company’s lead GalXC™ product candidate. [Press release from Dicerna™ Pharmaceuticals, Inc. discussing research presented at the German Society of Pediatric Nephrology 50^th Annual Meeting, Cologne] Press Release

Sensei Biotherapeutics Presents First Preclinical Data on SNS-723 CAR-T and LongTerm Results on SNS-301 Data

Sensei Biotherapeutics, Inc. announced new data from two programs targeting a novel tumor specific embryonic antigen, human aspartate β-hydroxylase (ASPH), including preclinical data on SNS-723, a first-in-class CAR-T cell therapy demonstrating effective killing of tumor cells by a series of ASPH-specific CAR-Ts as well as additional data from its Phase I study on the long-term effects of SNS-301, a first-in-class cancer immunotherapy demonstrating that all patients experienced dose-dependent and durable ASPH-specific immune responses. [Press release from Sensei Biotherapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Celularity Announces Results of Phase I Studies of PNK-007, an Allogeneic, Off-the-Shelf, Placental-Derived Cell Therapy

Celularity, Inc. announced results from two Phase I studies of PNK-007, an investigational allogeneic off-the-shelf natural killer (NK) cell therapy, in patients with acute myeloid leukemia and in patients with multiple myeloma. [Press release from Celularity, Inc. (Business Wire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

F1 Oncology to Present Data that Expands on Proof of Novel Point of Care Approach for CAR-T Therapies in Solid Tumor Malignancies

F1 Oncology, Inc. will present four abstracts that support novel technologies to treat solid tumor malignancies. [Press release from F1 Oncology, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Oncorus Presents Preclinical Data Supporting the Advancement of Its Lead Oncolytic Virus Therapy Clinical Candidate, ONCR-177

Oncorus, Inc. unveiled preclinical data yesterday supporting the clinical advancement of its lead oncolytic virus candidate, ONCR-177, during an oral presentation. [Press release from Oncorus, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

IO Biotech Presents Data on an Immunomodulatory CCL22-Based Vaccine

IO Biotech presented preclinical data on the therapeutic potential of an immunomodulatory CCL22-based vaccine. [Press release from IO Biotech (PR Newswire Association LLC) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Torque Presents New Preclinical Data for Its Deep-Primed Cellular Immunotherapy Programs

Torque announced two preclinical data presentations for its cellular immunotherapy programs. [Press release from Torque discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Fate Therapeutics Presents Late-Breaking Preclinical Data Highlighting Unique Advantages of Clonal Master Engineered iPSC Lines for Off-the-Shelf CAR T-Cell Therapy

Fate Therapeutics, Inc. presented late-breaking preclinical data highlighting the company’s unmatched ability to mass produce uniformly engineered chimeric antigen receptor (CAR) T cells for off-the-shelf cancer immunotherapy. [Press release from Fate Therapeutics, Inc. discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Allogene Therapeutics Presents Preclinical Data Demonstrating the Potential of AlloCAR T™ Therapy in Renal Cell Carcinoma (RCC)

Allogene Therapeutics, Inc. announced results from a preclinical study of its AlloCAR T program targeting CD70, a cancer target that is expressed on both hematologic and solid tumor cells. [Press release from Allogene Therapeutics, Inc. (GlobeNewswire, Inc.) discussing research presented at the American Association for Cancer Research (AACR) 2019 Annual Meeting, Atlanta] Press Release

Ichor Medical Systems Announces Collaboration and Research License Agreement with AstraZeneca to Develop DNA-Based Monoclonal Antibody Platform

Ichor^® Medical Systems, Inc. announced that it has entered into a collaboration and research license agreement with AstraZeneca for the development and clinical assessment of plasmid DNA constructs. [Ichor^® Medical Systems, Inc. (Business Wire, Inc.)] Press Release

TCR² Therapeutics Announces Collaboration with Cell and Gene Therapy Catapult for the Manufacturing of Its Novel T Cell Therapies for Cancer

TCR²Therapeutics Inc. announced it has selected the advanced therapy medicinal products ecosystem in the United Kingdom to establish a site for global manufacturing systems and capabilities. [TCR²Therapeutics Inc.] Press Release

StrideBio and Takeda Sign Collaboration and License Agreement to Advance Novel Gene Therapies for Neurological Diseases

StrideBio, Inc announced the signing of a collaboration and license agreement with Takeda Pharmaceutical Company Limited to develop in vivo adeno-associated viral based therapies for Friedreich’s Ataxia and two additional undisclosed targets. [StrideBio, Inc] Press Release

Phio and Glycostem to Collaborate on Use of Its sd-rxRNA^® platform and Glycostem’s oNKord^® Cell Therapy Products for the Next Generation of Natural Killer Cell-Based Immunotherapy for Cancer Treatment

Phio Pharmaceuticals Corp. announced that it has entered into a research collaboration with Glycostem Therapeutics BV to explore the potential synergies of using Phio’s self-delivering RNAi technology in combination with Glycostem’s proprietary Natural Killer-cell generation technology to develop cellular immunotherapies for cancer treatment with enhanced efficacy and/or safety. [Phio Pharmaceuticals Corp.] Press Release

Harvard Researchers’ Immune-Silent Stem Cell Technology Licensed to Sana Biotechnology

Harvard’s Office of Technology Development has executed a license agreement with Sana Biotechnology, a recently launched company co-founded by Harvard faculty that is focused on creating and delivering engineered cells as medicines for patients. Harvard’s foundational technology consists of methods for producing hypoimmunogenic stem cells that can be differentiated into any cell type and then transplanted into a patient without triggering immune rejection. [Harvard Office of Technology Development] Press Release

Orgenesis Signs Master Service and Joint Venture Agreement with TheraCell Advanced Biotechnology for the Development of Cell and Gene Therapies

Orgenesis Inc. announced that it has entered into a Master Service and Joint Venture Agreement with TheraCell Advanced Biotechnology, for the clinical development and commercialization of cell and gene therapies. [Orgenesis Inc.] Press Release

Oxford Biomedica Notes the Japanese Approval of Kymriah^® (Tisagenlecleucel), the First CAR-T Cell Therapy Authorized in Asia

Oxford Biomedica plc noted an announcement by Novartis that Japan’s Ministry of Health, Labor and Welfare has approved Kymriah^® for the treatment of two distinct indications – CD19-positive relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia and CD19-positive r/r diffuse large B-cell lymphoma. [Oxford Biomedica plc] Press Release

Fibrocell Announces Positive Feedback from Type B End-of-Phase II Meeting with FDA on Phase III Clinical Trial Design for FCX-007

Fibrocell Science, Inc. announced the completion of a Type B end-of-Phase II meeting with the FDA to discuss the design of a Phase III clinical trial for FCX-007, the company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa, to support a Biologics License Application filing. [Fibrocell Science, Inc.] Press Release

Herantis Pharma Announces Expansion of Its Phase II Study AdeLE in Breast Cancer Associated Lymphedema with Two Centers in Sweden

Herantis Pharma Plc announced the expansion of its Phase II AdeLE clinical trial in Sweden to include the Karolinska University Hospital in Stockholm, and the Uppsala University Hospital. [Herantis Pharma Plc] Press Release

bluebird bio Receives Positive Opinion from CHMP for ZYNTEGLO™ (Autologous CD34⁺ Cells Encoding βA-T87Q-Globin Gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia (TDT) Who Do Not Have β0/β0 Genotype

bluebird bio, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™, a gene therapy for patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. [bluebird bio, Inc.] Press Release

Incysus Therapeutics Announces FDA Approval of IND Application for a Novel Gamma-Delta (γδ) T Cell Therapy for Treatment of Patients With Newly-Diagnosed Glioblastoma

Incysus Therapeutics, Inc. announced that the FDA has approved the Investigational New Drug (IND) application for the clinical study of a genetically modified γδ T cell in combination with chemotherapy for the treatment of patients with newly-diagnosed glioblastoma. [Incysus Therapeutics, Inc. (GlobeNewswire, Inc.)] Press Release

NantKwest Announces New haNK Natural Killer Cell Patent Issuance Further Expanding Intellectual Property Estate

NantKwest announced that the company has been issued a new patent that further expands the company’s foundational natural killer cell-therapeutics platform intellectual property estate. [NantKwest] Press Release

First Gene Therapy to Treat Rare Blood Disease Nears European Approval

The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use, the drug-reviewing arm of the European Medicines Agency. [STAT News] Editorial

Recommendations for Regulating the Environmental Risk of Shedding for Gene Therapy and Oncolytic Viruses in Canada

Canadian academic and industry stakeholders are concerned about the inclusion of “virus-like particles or sub-viral particles” in the definition of New Substances Notification Regulations for Organisms (NSNR(O)) which impacts clinical cell and gene therapy and commercialization. [Front Med] Article

What Thailand’s Long-Awaited Election Could Mean for Science

Thailand is bracing for the results of its first election since a military junta took charge in a 2014 coup. Scientists, like many other people in Thailand, want a more democratic country. But whether a pro-democracy party or a military-backed one ends up in charge, science is likely to prosper as a result of ongoing reforms put in place by the junta that are likely to continue under either government. [Nature News] Editorial

National Academy of Sciences Will Vote on Ejecting Sexual Harassers

The U.S. National Academy of Sciences in Washington, D.C., will ask its members this month to change the organization’s bylaws to allow proven sexual harassers and those guilty of other misconduct to be ejected from their ranks. That’s a first for the prestigious organization that advises the U.S. government on scientific issues: Its members, who are voted in by other members, have always been elected for life. [ScienceInsider] Editorial

FDA

Advisory Committee; Cellular, Tissue and Gene Therapies Advisory Committee, Renewal (FR Doc. No:2019-05985) Notice

NEW 24^th Congress of the European Hematology Association (EHA)
June 13-16, 2019
Amsterdam, Netherlands

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

NEW Director of Research – Division of Translational Medicine & Human Genetics (University of Pennsylvania)

NEW Staff Scientist – Development of Mechanism-Based Tumor Therapies (Frankfurt Cancer Institute)

Research Technologist – Immunology (STEMCELL Technologies Inc.)

Research Associate – Bioengineering (STEMCELL Technologies Inc.)

Postdoctoral Position – Immunotherapy (McGill University)

Postdoctoral Research Associate – Retinal Stem Cell Biology and Regeneration (University of Nebraska Medical Center)

Cell Biologist – Regenerative Medicine (Hepatx Corporation)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

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

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)

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

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

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

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