GSK promises ‘bubble boy’ gene therapy will not break the bank as new pricing model revealed

Apr 6, 2016

GlaxoSmithKline ($GSK) has only just received a thumbs-up from a European Medicines Agency committee for its groundbreaking new gene therapy treatment–but it’s already thinking about pricing as it gears up for a final European approval in the coming months. The CHMP–the safety and efficacy arm of the European regulator–recommended approval of GSK’s new drug Strimvelis on Friday to treat the incredibly rare and fatal disorder known colloquially as “bubble boy syndrome,” more than a decade after the EMA first granted the drug an orphan designation. Strimvelis has been developed in partnership with Italy’s San Raffaele Telethon Institute for Gene Therapy and works as an ex vivo gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)–aka “bubble boy syndrome,” as children with the disorder must be kept protected from any infections due to their highly fragile immune system. ...

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Quantitative Systems Pharmacology Modeling (QSPM) Case Study in Immuno-oncology

Apr 6, 2016

DATE: Tuesday, April 12, 2016 | TIME: 11am ET / 8am PT | DURATION: 1 Hour Biotech and Pharma are increasingly using quantitative systems pharmacology modeling (QSPM) approaches to help answer complex, critical Go/No-go decisions in R&D. Dr. John Burke will discuss the: Value of QSP approaches A QSP industry case study The case study entails developing and interrogating a QSPM of PD-1 and TIM-3. The QSPM provided: Biological insights by predicting why dosing regimens are quite similar for two therapeutics targeting PD-1 that have affinities that differ by several orders of magnitude Best-in-class profile predictions for PD-1 and TIM-3 dual antagonists This webinar is ideal for scientists in R&D who want to learn more about how to leverage QSPM to shorten timelines, lower cost, and increase the likelihood of developing best-in-class drugs. FEATURED SPEAKER: John M. Burke, PhD — Co-Founder, President, and CEO, Applied BioMath Prior to co-founding Applied BioMath, Dr. Burke was an Associate Director, Head of Systems Biology at Boehringer Ingelheim where he started, developed and managed... ...

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Webinar: Minimizing Risk to Maximize Coverage for the Global Supply of Vaccines

Apr 6, 2016

This presentation will focus on the use of the advanced aseptic filling techniques utilized in blow/fill/seal technology to enhance the global supply of vaccines through primary container design and manufacturing efficiency. In this webinar, we will discuss: the details and data on how the principles of BFS dramatically reduce the risk profile and particulate contamination in aseptic filling. information on the container closure and a case study of a model monoclonal antibody in a BFS vial compared to glass. primary container design freedom to help address the needs of vaccine distribution and delivery. FEATURED SPEAKER:   Bill Hartzel — Director, Strategic Execution, Advanced Delivery Technologies, Catalent Pharma Solutions Bill Hartzel is the Director of Strategic Execution at Catalent Pharma Solutions, a global leader in development solutions and advanced drug delivery technologies. In this role, he is responsible for the implementation of a full suite of advanced aseptic processing solutions for biologic and complex pharmaceutical liquid products. He provides a strong background in advanced aseptic processing in blow/fill/seal and plastics in... ...

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Why Should Certainly Collect Hockey Jerseys

Jun 9, 2013

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