Cell Therapy News Volume 20.41 | Dec 9 2019

TOP STORY

CAR T-Cells Targeting Müllerian Inhibiting Substance Type 2 Receptor for the Treatment of Ovarian Cancer and other Gynecologic Malignancies

The authors describe the development and evaluation of a CAR that targets the Müllerian inhibiting substance type 2 receptor (MISIIR). T-cells expressing the MISIIR-specific CAR demonstrated antigen-specific reactivity in vitro and eliminated MISIIR-overexpressing tumors in vivo. [Mol Ther] Abstract

(Ranked by impact factor of the journal)

Targeted Gene Therapy using AAV1.Serca2a Reverses Pulmonary Fibrosis by Blocking the STAT3/FOXM1 Pathway and Promoting the SNON/SKI Axis

Investigators found that SERCA2a expression was significantly decreased in lung samples from patients with pulmonary fibrosis (PF) and in the bleomycin (BLM) mouse model of PF. In the BLM-induced PF model, intratracheal aerosolized adeno-associated virus serotype 1 (AAV1) encoding for human SERCA2a reduced lung fibrosis and associated vascular remodeling. [Mol Ther] Abstract | Graphical Abstract

CircRNA hsa_circ_0074834 Promotes the Osteogenesis-Angiogenesis Coupling Process in Bone Mesenchymal Stem Cells (BMSCs) by Acting as a ceRNA for miR-942-5p

After isolation of bone-marrow mesenchymal cells (BMSCs) from patients with nonunion, the expression of circRNAs in these cells was detected by using a circRNA microarray. Alkaline phosphatase and Alizarin red staining were used to detect the regulation of osteogenic differentiation of BMSCs by hsa_circ_0074834. [Cell Death Dis] Full Article

Short and Long Term Clinical and Immunologic Follow Up After Bone Marrow Mesenchymal Stromal Cell Therapy in Progressive Multiple Sclerosis- a Phase I Study

Scientists conducted aPphase I clinical study assessing safety and clinical and peripheral immune responses after mesenchymal stem cell (MSC) therapy in Multiple Sclerosis (MS). Seven patients with progressive MS were intravenously infused with a single dose of autologous MSC. The infusions were safe and well tolerated when given during clinical remission. [J Clin Med] Full Article

Preclinical Toxicity Evaluation of Clinical Grade Placenta-Derived Decidua Stromal Cells

Human placenta-derived decidua stromal cells (DSCs) were cultured and expanded from fetal membranes obtained from placentas following cesarean section. In rats, 0.5×10⁶ cells/kg were injected intravenously or intra-aortal. In mice, DSCs were given intravenously at doses ranging from 4–40×10⁶ cells/kg. [Front Immunol] Full Article

Alveolar Bone Repair of Rhesus Monkeys by Using BMP-2 Gene and Mesenchymal Stem Cells Loaded Three-Dimensional Printed Bioglass Scaffold

Scientists evaluated the osteogenesis of alveolar bone defects of animal in the following experimental groups: sham-operated, 3D printed bioglass (3D-BG), 3D-BG with BMP-2 gene loaded CS and 3D-BG with rhesus marrow bone mesenchymal stem cells and BMP/CS.” [Sci Rep] Full Article

Variation in Zygotic CRISPR/Cas9 Gene Editing Outcomes Generates Novel Reporter and Deletion Alleles at the Gdf11 Locus

Investigators report the use of zygotic CRISPR/Cas9 injections to generate a knock-in GFP reporter mouse at the Gdf11 locus. Phenotypic and genomic characterization of founder animals from these injections revealed a subset that contained the correct targeting event and exhibited GFP expression that, within the hematopoietic system, was restricted predominantly to lymphoid cells. [Sci Rep] Full Article

PDGFB-Expressing Mesenchymal Stem Cells Improve Human Hematopoietic Stem Cell Engraftment in Immunodeficient Mice

Researchers compared the effects of EGF, FGF2, and PDGFB on HSC regeneration using human mesenchymal stem cells that were transduced with these factors via lentiviral vectors. Among the above niche factors tested, MSCs transduced with PDGFB most significantly improved human HSC engraftment in immunodeficient mice. [Bone Marrow Transplant] Full Article

Nitinol Thin Films Functionalized with CAR-T Cells for the Treatment of Solid Tumors

In a mouse model of non-resectable ovarian cancer, cell-loaded nitinol thin films spatially conformed to the implantation site, fostered the rapid expansion of T cells, delivered a high density of T cells directly to the tumor and significantly improved animal survival. [Nat Biomed Eng] Abstract

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T_reg Cell-Based Therapies: Challenges and Perspectives

Investigators discuss the biology of T_reg cells and describe new efforts in T_reg cell engineering to enhance specificity, stability, functional activity and delivery. They envision that the success of T_reg cell therapy in autoimmunity and transplantation will encourage the clinical use of adoptive T_reg cell therapy for non-immune diseases, such as neurological disorders and tissue repair. [Nat Rev Immunol] Abstract

Human Germline Genome Editing

Unresolved questions about pre-implantation human development could be addressed by basic research using CRISPR-Cas9. The authors discuss advances in human genome editing and consider ethical questions and potential clinical implications of this technology. [Nat Cell Biol] Abstract

Role of Liposomes-Based Stem Cell for Multimodal Cancer Therapy

Scientists highlight and summarize the mechanism involved in stem cell migration, strategies to enhance homing, safety and efficacy studies of stem cells in various disease models and discuss the potential role of liposomes in prolonged and localized delivery of bioactives for regenerative medicines and tissue engineering techniques. [Stem Cell Rev Rep] Abstract | Graphical Abstract

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Ocugen Announces Completion of 50% of Enrollment of Its Phase III Clinical Trial for Ocular GVHD

Ocugen, Inc. announced that it has completed 50% of enrollment of its Phase III clinical trial for OCU300 for patients with ocular Graft Versus Host Disease (oGVHD). Ocugen is the first and only company to receive Orphan Drug Designation for a product candidate designed to treat oGVHD and is the first company to conduct a Phase III clinical trial in patients with this disease. [Ocugen, Inc.] Press Release

Abeona Therapeutics Cleared to Initiate Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

Abeona Therapeutics Inc. announced that the FDA has removed its clinical hold and provided clearance to proceed with the VIITAL™ study, the company’s pivotal Phase III clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa. [Abeona Therapeutics Inc.] Press Release

Bristol-Myers Squibb and Bluebird Bio Announce Positive Top-Line Results From the Pivotal Phase II KarMMa Study of Ide-Cel in Relapsed and Refractory Multiple Myeloma

Bristol-Myers Squibb Company and bluebird bio, Inc. announced positive top-line results from KarMMa, a pivotal, open-label, single arm, multicenter, Phase II study of idecabtagene vicleucel. KarMMa, which evaluated the efficacy and safety of the companies’ led investigational BCMA-targeted chimeric antigen receptor T cell therapy candidate for patients with relapsed and refractory multiple myeloma, met its primary endpoint and key secondary endpoint. [Bristol-Myers Squibb Company] Press Release

Janssen Presents Initial Results for BCMA CAR-T Therapy JNJ-4528 Showing Early, Deep and High Responses in the Treatment of Relapsed or Refractory Multiple Myeloma

The Janssen Pharmaceutical Companies of Johnson & Johnson announced initial results from the Phase Ib/II CARTITUDE-1 study evaluating the efficacy and safety of JNJ-68284528, an investigational B cell maturation antigen (BCMA)-directed CAR-T therapy being evaluated in the treatment of patients with relapsed or refractory multiple myeloma. [The Janssen Pharmaceutical Companies (BusinessWire, Inc.)] Press Release

Rocket Pharmaceuticals Announces Preliminary Data from Phase I/II Trial of RP-L201 for Leukocyte Adhesion Deficiency-I

Rocket Pharmaceuticals, Inc. announced preliminary data from its Phase I/II clinical trial of RP-L201, the Company’s lentiviral vector-based gene therapy for the treatment of Leukocyte Adhesion Deficiency-I. [Rocket Pharmaceuticals, Inc.] Press Release

CARsgen Therapeutics Receives IND Clearance From the NMPA for AB011 Humanized Claudin18.2 Monoclonal Antibody

CARsgen Therapeutics announced that one of its leading drug candidates, AB011 humanized claudin18.2 monoclonal antibody for the treatment of patients suffering from advanced gastric and pancreatic adenocarcinoma, has received Investigational New Drugclearance from the National Medical Products Administration in China. [CARsgen Therapeutics Co. Ltd. (PR Newswire Association LLC.)] Press Release

Tufts to Remove Sackler Name from Medical Campus

Tufts University will not return donations from the family that made its riches on opioids, but the university will start a $3 million endowment for addiction prevention and treatment. [The Scientist] Editorial

Merck and Sanofi Buy Smaller Cancer Drug Firms at Big Premiums, Fueling Investor Excitement

The drug giants Merck and Sanofi each said they would acquire a smaller drug maker for more than $2 billion – in each case, more than double the smaller company’s market capitalization – cheering investors about large companies’ appetite to execute buyouts in the biotechnology sector. [STAT News] Editorial

House Republicans Fuel Capitol Hill Chaos with Competing Drug Pricing Bill

Republicans are set to accelerate Capitol Hill’s drug pricing chaos with a new proposal to lower the cost of prescription medicines, according to a summary document obtained by STAT. [STAT News] Editorial

NEW Keystone Symposia: Noncoding RNAs – Mechanism, Function and Therapies
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