Gene Therapy Briefs

U.S. Food and Drug Administration. FDA Briefing Document. BLA 125646: Tisagenlecleucel, Novartis Pharmaceuticals Corporation. FDA Oncologic Drugs Advisory Committee Meeting, 2017. Available at: www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM566166.pdf (Accessed August 9, 2017).

Novartis.. Novartis CAR-T cell therapy CTL019 unanimously (10-0) recommended for approval by FDA advisory committee to treat pediatric, young adult r/r B-cell ALL. July 13, 2017. Available at: www.novartis.com/news/media-releases/novartis-car-t-cell-therapy-ctl019-unanimously-10-0-recommended-approval-fda (Accessed August 9, 2017).

Spark Therapeutics.. Spark Therapeutics submits Marketing Authorization Application to European Medicines Agency for investigational LUXTURNA™ (voretigene neparvovec). July 31, 2017. Available at: http://ir.sparktx.com/phoenix.zhtml?c=253900&p=irol-newsArticle&ID=2290110 (Accessed August 9, 2017).

GlaxoSmithKline.. GSK delivers further progress in Q2 and sets out new priorities for the Group. July 26, 2017. Available at: https://gsk.com/en-gb/media/press-releases/gsk-delivers-further-progress-in-q2-and-sets-out-new-priorities-for-the-group/ (Accessed August 10, 2017).

Mullin E. A year after approval, gene-therapy cure gets its first customer. MIT Technology Review May 3, 2017. Available at: www.technologyreview.com/s/604295/a-year-after-approval-gene-therapy-cure-gets-its-first-customer (Accessed August 10, 2017).

Sagonowsky E. GlaxoSmithKline's talk of leaving rare diseases—amid slow Strimvelis sales—highlights challenges in the field. FiercePharma July 27, 2017. Available at: www.fiercepharma.com/pharma/gsk-s-talk-leaving-rare-diseases-highlights-challenges-field (Accessed August 10, 2017).

Oregon Health and Science University.. Study in Nature demonstrates method for repairing genes in human embryos that prevents inherited disease. AAAS/EurekAlert August 2, 2017. Available at: www.eurekalert.org/pub_releases/2017-08/ohs-sin080217.php (Accessed August 9, 2017),

Ma H. , Marti-Gutierrez N. , Park S. , et al. Correction of a pathogenic gene mutation in human embryos. Nature 2017 Aug 2 [Epub ahead of print] Doi:10.1038/nature23305. Available at: www.nature.com/nature/journal/vaop/ncurrent/full/nature23305.html?foxtrotcallback=true#discussion (Accessed August 9, 2017).

uniQure.. uniQure reacquires development and commercialization rights for its gene therapy candidate in Hemophilia B. July 31, 2017. Available at: https://tools.eurolandir.com/tools/Pressreleases/GetPressRelease/?ID=3362062&lang=en-GB&companycode=nl-qure&v= (Accessed August 9, 2017).

Alexion Pharmaceuticals. Form 10-K annual report for the fiscal year ended December 31, 2016. Filed February 16, 2017. Available at: http://ir.alexionpharm.com/secfiling.cfm?filingID=899866-17-44&CIK=899866 (Accessed August 9, 2017).

Alexion Pharmaceuticals.. Alexion reports second quarter 2017 results. July 27, 2017. Available at: http://news.alexionpharma.com/press-release/financial-news/alexion-reports-second-quarter-2017-results (Accessed August 9, 2017).

Moderna Therapeutics.. Moderna provides update on mRNA rare disease R&D strategy. July 27, 2017. Available at: www.modernatx.com/newsroom/press-releases/moderna-provides-update-mrna-rare-disease-rd-strategy (Accessed August 9, 2017).