Genetic Engineering & Biotechnology News

AUG 2013

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D; community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Drug Discovery Hematologic Diagnostics Go with the Flow Thalyana Smith-Vikos Those diagnosing hematopathological disorders have been reaping the benefts of improved fow cytometry methodologies, though standardization of some techniques is still in the works. Flow cytometry is being used to identify and evaluate clinical cases of various blood disorders. Researchers have also adapted other types of cellular analyses, such as cytogenetics and molecular genetics, in order to monitor disease states. Anna Porwit, M.D., Ph.D., at the University of Toronto, has been immunophenotyping lymphoproliferative disorders with more than one malignant cell population. To investigate these diseases, Dr. Porwit and her colleagues use fow cytometry, which is a common immunophenotyping method in hematopathological diagnosis, to identify aberrant populations of cells in the blood and bone marrow. As medical director of the university's fow cytometry lab, Dr. Porwit instituted a 10-color fow cytometry method, using the Navios™ instrument from Beckman Coul- ter. Navios, which contains three lasers, allows Dr. Porwit and her colleagues to analyze 10 different antibodies simultaneously on the same cell. By using monoclonal antibodies with different connected fuorochromes, 10 different antibodies can be assayed in one tube. To study lymphoid malignancies, Dr. Porwit utilized two 10-color tubes of surface markers for B- and T-lymphocytes, respectively. She then evaluated kappa/lambda ratios separately in various B-cell subsets. Dr. Porwit noticed that in some lymphoma cases, there were two different pathological populations of B or T cells in one sample, which was quite unusual because typically there is only a single aberrant population of cells. In fact, Dr. Porwit and her colleagues identifed a cohort of cases where there were Upper panel dotplot 3 shows normal maturation of CD10 and CD20 on B cells going from blue to light green and then fully mature are dark green. Bottom panel shows an additional population in the "negative space" on dotplot 6 compared to dotplot 3. The abnormal population represents 0.08% of CD45+ cells. London Health Sciences Centre See Hematologic Dx on page 18 NEWS Discovery & Development > Immunocore Teams Up with nocore in development and commercial milestone payments, plus up to doubledigit royalties. Genentech, GSK in Two Separate ImmTAC Deals University of Oxford spinout Immunocore has entered into two separate deals with biotech and pharma giants based on its ImmTAC technology, which together could potentially net the firm upwards of $808 million. The first deal, with Genentech, centers on the discovery and development of multiple cancer targets using ImmTACs— bispecific biological drugs that make use of the power of T-cell receptors to recognize intracellular changes that can occur during viral infections and cancer. Under the terms of the agreement, Immunocore is slated to receive an initiation fee of between $10 million and $20 million, plus in excess of $300 million in development and commercial milestone payments for each target program as well as tiered royalties. Under the terms of a second agreement, which Immucore has inked with GlaxoSmithKline, the firms will use ImmTACs for multiple targets not addressable using antibody-based technologies. 16 | > Forma, Cancer Research UK in DUBs Drug Discovery Collaboration T cell (grey) killing a tumor cell (yellow) Immunocore Immunocore will be responsible for all of the preclinical development and for initial clinical trials, while GSK will be responsible for the remaining development and commercialization of the products. Per this deal, Immunocore is eligible to receive up to a total of £142 million (approximately $211 million) in preclinical milestone payments from GSK across the targets. In addition, for each product that reaches the market, up to £200 million (around $297.3 million) is due to Immu- August 2013 | GENengnews.com | Genetic Engineering & Biotechnology News Forma Therapeutics and Cancer Research UK subsidiary Cancer Research Technology (CRT) are together working to discover and develop tools, technologies, and therapeutic candidates against a variety of deubiquitinating enzymes, or DUBs. Under the terms of the agreement, Forma will pair its drug discovery capabilities with CRT's technology transfer expertise and network of Cancer Research UK investigators. Together, the organizations aim to investigate the protease enzymes that regulate ubiquitin-dependent pathways implicated in cancer. > European Consortium that Aims to Develop S. Aureus Vaccine Gets $7M Boost Scientists from French biopharma firm Imaxio, Oxford University's Jenner Institute, the European Vaccine Initiative, and Swiss CRO Preclin Biosystems have won a €5.5 million (approximately $7 million) grant to support the Bellerophon Project consortium, which aims to develop a vaccine against Staphylococcus aureus. The funds will support preclinical testing and a Phase I clinical trial, slated to begin in 2016, the organizations said. > RaNA Taps Santaris LNA Chemistry for RNA-Targeted Drug Development RaNA Therapeutics and Santaris Pharma are working together to develop RNA-targeted therapeutics that selectively activate protein expression with an initial focus on rare genetic diseases, inflammation, cancers, metabolic diseases, and neurodegenerative diseases, the companies said. Under the terms of the agreement, RaNA has licensed rights to use Santaris' Locked Nucleic Acid (LNA) Drug Platform. RaNA scientists will be using this technology for the development of LNA-based drug candidates against up to 10 of its own RNA targets. n

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