Volume 16.17 | May 4

Issue 16.17 May 4, 2015
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Study Points Way Forward for Retinal Disease Gene Therapy
Gene therapy for Leber congenital amaurosis, an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment. Both of these benefits, however, peaked one to three years after treatment and then diminished, according to results from an ongoing clinical trial. [Press release from the National Institutes of Health discussing online prepublication in The New England Journal of Medicine] Press Release | Abstract
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PUBLICATIONS (Ranked by impact factor of the journal)
Immunosuppressive Plasma Cells Impede T-Cell-Dependent Immunogenic Chemotherapy
Scientists showed that mouse B cells modulate the response to low-dose oxaliplatin, which promotes tumor-directed cytotoxic T cells activation by inducing immunogenic cell death. [Nature] Abstract | Press Release

Allogeneic IgG Combined with Dendritic Cell Stimuli Induce Antitumor T-Cell Immunity
The authors found that allogeneic tumor rejection is initiated in mice by naturally occurring tumor-binding IgG antibodies, which enable dendritic cells to internalize tumor antigens and subsequently activate tumor-reactive T cells. They exploited this mechanism to treat autologous and autochthonous tumors successfully. [Nature] Abstract | Press Release

CMV-Specific T Cells Generated from Naïve T Cells Recognize Atypical Epitopes and May Be Protective In Vivo
Researchers demonstrated the feasibility of deriving cytomegalovirus (CMV)-specific T cells from naïve cells for T cell therapy. When infused to recipients, naïve donor–derived virus-specific T cells that recognized atypical epitopes were associated with prolonged periods of CMV-free survival and complete remission. [Sci Transl Med] Abstract | Press Release

Cloning-Free CRISPR/Cas System Facilitates Functional Cassette Knock-In in Mice
Scientists showed that cloning-free, direct nuclear delivery of Cas9 protein complex with chemically synthesized dual RNAs enables highly efficient target digestion, leading to generation of knock-in mice carrying a functional cassette with up to 50% efficiency, compared with just 10% by a commonly used method consisting of Cas9 mRNA and single guide RNA. [Genome Biol] Full Article | Press Release

Down-Regulating HIF-1α by Lentivirus-Mediated shRNA for Therapy of Triple Negative Breast Cancer
Investigators designed a recombinant lentivirus containing hypoxia inducible factor 1α (HIF-1α) siRNA, developed stably transfected cell lines, and tested the anticancer effects of the siRNA on cancer cells in vitro and in vivo. [Cancer Biol Ther] Abstract

211Astatine-Conjugated Monoclonal CD45 Antibody-Based Nonmyeloablative Conditioning for Stem Cell Gene Therapy
Scientists demonstrated the feasibility of using a nonmyeloablative dose of a monoclonal anti-CD45 antibody conjugated to the alpha-emitter astatine-211 to promote engraftment of an autologous gene-modified stem cell graft in the canine model. [Hum Gene Ther] Abstract

Autologous Transplantation of Oral Mucosal Epithelial Cell Sheets Cultured on an Amniotic Membrane Substrate for Intraoral Mucosal Defects
Researchers investigated the autologous transplantation of oral mucosal epithelial cells cultured on amniotic membrane in patients undergoing oral surgeries. Five patients without any history of a medical disorder who underwent autologous cultured oral epithelial transplantation following oral surgical procedures were included. [PLoS One] Full Article

Arthroscopic Transplantation of Synovial Stem Cells Improves Clinical Outcomes in Knees with Cartilage Defects
Investigators aimed to determine whether transplantation of synovial mesenchymal stem cells improve MRI features, histologic features and clinical evaluation scores in patients with cartilage defects in the knee. [Clin Orthop Relat Res] Abstract | Full Article

Treatment of Macular Degeneration Using Embryonic Stem Cell-Derived Retinal Pigment Epithelium: Preliminary Results in Asian Patients
Researchers report the safety and tolerability of subretinal transplantation of human embryonic-stem-cell-derived retinal pigment epithelium in four Asian patients: two with dry age-related macular degeneration and two with Stargardt macular dystrophy. [Stem Cell Reports] Full Article | Press Release

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Mesenchymal Stromal Cell Therapy in Hematology: From Laboratory to Clinic and Back Again
The authors focus on cell therapy applications for mesenchymal stromal cells (MSC) in hematology. In this domain, MSC are used for the treatment or prevention of graft versus host disease, support of hematopoiesis, or repair of tissue toxicities after hematopoietic cell transplantation. They critically review the accumulating clinical data and elaborate on complications that might arise from treatment with MSC. [Stem Cells Dev] Abstract

Immune-Related Strategies Driving Immunotherapy in Breast Cancer Treatment: A Real Clinical Opportunity
The authors briefly describe the most recently awarded immune-based therapies with a consolidated or potential implication for the treatment of solid malignancies. They focus on immune checkpoints and on the clinical potential of their abrogation, with a further overview of novel vaccine-based approaches and the most relevant immunotherapeutic techniques. [Expert Rev Anticancer Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research fields.

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Derma Sciences Presents Clinical Data Demonstrating Efficacy in Tissue Regeneration
Derma Sciences, Inc., a leading tissue regeneration company at the forefront of research and innovation for the management of acute and chronic wounds and burns, presented 14 posters on four of the company’s products related to tissue regeneration in advanced wound care. [Press release from Derma Sciences, Inc. discussing research presented at the Spring Symposium on Advanced Wound Care (SAWC), San Antonio] Press Release

Spark Therapeutics to Have Multiple Presentations
In its development of SPK-RPE65 as a potential gene therapy treatment for RPE65-mediated inherited retinal degenerations, Spark is utilizing a proprietary mobility test of functional vision as the primary endpoint in its current Phase III clinical trial. [Press release from Spark Therapeutics, Inc. (GlobeNewswire, Inc.) discussing research presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO), Denver] Press Release

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NIH Reiterates Ban on Editing Human Embryo DNA
The US National Institutes of Health (NIH) has reaffirmed its ban on research that involves gene editing of human embryos. In a statement, NIH director Francis Collins spelled out the agency’s long-standing policy against funding such research and the ethical and legal reasons for it. [Nature News] Editorial

New Version of Cures Bill Recommends $10 Billion Boost for NIH
An ambitious effort in the U.S. House of Representatives to jump-start biomedical innovation took another step forward with the release of a bipartisan draft bill. The so-called 21st Century Cures Act contains huge news for supporters of biomedical research: It recommends substantial budget increases for the National Institutes of Health (NIH), including $10 billion in extra funding over five years. [ScienceInsider] Editorial

US Clinics Avoiding Government Oversight of ‘Stem Cell’ Treatments
Clinics across the United States are advertising stem cell treatments that attempt to take advantage of what they perceive as exceptions in FDA regulations, according to bioethicist Leigh G. Turner. The therapies in question are adipose-derived autologous stem cell treatments, in which fat cells are removed from a patient, broken down to separate components that purportedly contain stem cells, and are then reinjected into the same patient. [Elsevier B.V.] Press Release | Commentary
4D Molecular Therapeutics and Roche Sign Collaboration Agreement for Gene Therapy Vector Discovery and Product Development
4D Molecular Therapeutics announced a collaboration and license agreement with Roche to discover and develop optimized next-generation AAV vectors for indications with high unmet medical need. [4D Molecular Therapeutics] Press Release

Cell Therapy Catapult and Asymptote Collaborate to Revolutionize Delivery of Cell Therapies
The Cell Therapy Catapult and Asymptote are combing forces to advance the development of a novel point-of-care thawing system for cell-based therapies. This device is intended to solve a global barrier to commercialization of cell therapies, by simplifying the delivery of these therapies to patients by addressing significant challenges of delivery at the point-of-care. [Cell Therapy Catapult] Press Release

Spark Therapeutics and Clearside Biomedical Announce Exclusive Option to License Technology for Potentially Differentiated Delivery of Gene Therapy to the Eye
Spark Therapeutics, Inc. and Clearside Biomedical, Inc. announced that they have entered into an option agreement under which Spark acquired exclusive rights to license Clearside’s microinjector technology to deliver gene therapies to the back of the eye. [Spark Therapeutics, Inc.] Press Release

Bellicum Pharmaceuticals Licenses TCR Technology from Leiden University Medical Center
Bellicum Pharmaceuticals, Inc. announced it has entered into a license agreement with Leiden University Medical Center for worldwide rights to develop, manufacture and commercialize high-affinity TCR (T cell receptor) product candidates targeting solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. [Bellicum Pharmaceuticals, Inc.] Press Release

NeoStem Announces First Patient Randomized in Pivotal Phase III Melanoma Immunotherapy Clinical Trial
NeoStem, Inc. announced the randomization of the first patient in the Intus Phase III clinical trial investigating the efficacy of the company’s patient-specific targeted cancer immunotherapy candidate NBS20 in patients with stage III recurrent or stage IV metastatic melanoma. [NeoStem, Inc.] Press Release

Cancer Treatment Centers of America at Western Regional Medical Center Launches Phase II of Innovative ‘PembroPlus’ Clinical Trial
Cancer Treatment Centers of America® at Western Regional Medical Center has begun the Phase II portion of the first of six arms of this first-of-its-kind clinical trial, using a new immunotherapy treatment for patients with advanced small cell lung cancer, pancreatic cancer and connective-tissue cancers, including breast cancer. [Cancer Treatment Centers of America®] Press Release

Amgen to Discuss Details of the Biologics License Application for Talimogene Laherparepvec for Patients with Metastatic Melanoma
Amgen announced that the company discussed the data supporting the Biologics License Application for talimogene laherparepvec monotherapy for the treatment of patients with injectable regionally or distantly metastatic melanoma at a joint meeting of the U.S. Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee and Oncologic Drugs Advisory Committee. [Amgen Inc.] Press Release

U.S. Food and Drug Administration Accepts Supplemental Biologics License Application for Opdivo (Nivolumab) in Patients with Previously Untreated Advanced Melanoma
Bristol-Myers Squibb Company announced that the U.S. Food and Drug Administration has accepted for filing and review the supplemental Biologics License Application for Opdivo for the treatment of previously untreated patients with unresectable or metastatic melanoma. [Bristol-Myers Squibb Company] Press Release

Advaxis Enhances Lm-LLO Cancer Immunotherapy Intellectual Property Portfolio with Two USPTO Patents Addressing ADXS-PSA and ADXS-HER2
Advaxis, Inc., a cancer immunotherapy company, announced that the United States Patent and Trademark Office (USPTO) has granted U.S. Patent No. 9,012,141 with composition of matter claims covering the Advaxis product candidate, ADXS-PSA, and U.S. Patent No. 9,017,660 with methods of use claims covering the Advaxis product candidate, ADXS-HER2. [Advaxis, Inc.] Press Release
Center for Scientific Review Notice of Closed Meetings (FR Doc. No: 2015-10273)
Approval Letter – RAPLIXA
Food and Drug Administration (United States)

Biosimilars: Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009; Guidance for Industry; Availability (FR Doc. No: 2015-10064)

Guidance for Industry on Quality Considerations in Demonstrating Biosimilarity of a Therapeutic Protein Product to a Reference Product; Availability (FR Doc. No: 2015-10063)
NEW 13th International Conference on Tissue Engineering and Regenerative Medicine
July 20-21, 2015
Paris, France

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.
NEW Cell Therapy Production Scientist (Paramount Recruitment)

Scientist – Reprogramming and Pluripotent Stem Cell Biology (STEMCELL Technologies Inc.)

Scientist – Liver Cell Biology (STEMCELL Technologies Inc.)

Research Assistant – Exosome-Based Gene Therapy for Huntington’s Disease (University of Oxford)

Postdoctoral Fellow – Neural Tissue Engineering (Nanyang Technological University)

Director – Clinical Trial Operations (BioRestorative Therapies, Inc.)

Director – Cell Therapy Manufacturing (BioRestorative Therapies, Inc.)

Professor – Gene Therapy (University of Massachusetts)

Research Associate – Cell Culture Specialist (Tisch MS Research Center of New York)

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