Gene Therapy Briefs

Novartis (2019). Novartis announces AVXS-101 intrathecal study update. https://www.novartis.com/news/media-releases/novartis-announces-avxs-101-intrathecal-study-update (last accessed November 14, 2019).

AveXis, a Novartis Company (2019). AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma^® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) type 1. https://www.novartis.com/news/media-releases/avexis-presents-new-data-epns-continuing-show-significant-therapeutic-benefit-zolgensma-prolonging-event-free-survival-now-5-years-age-patients-spinal-muscular (last accessed November 14, 2019).

Solid Biosciences (2019). Solid biosciences provides SGT-001 program update. https://www.solidbio.com/about/media/press-releases/solid-biosciences-provides-sgt-001-program-update (last accessed November 14, 2019).

Wyss Institute for Biologically Inspired Engineering (2019). Combination gene therapy treats multiple age-related diseases in mice. https://wyss.harvard.edu/news/combination-gene-therapy-treats-multiple-age-related-diseases/ (last accessed November 15, 2019).

Davidsohn N , Pezzone M , Vernet A , et al. (2019) A single combination gene therapy treats multiple age-related diseases. PNAS. https://www.pnas.org/content/early/2019/11/08/1910073116 (last accessed November 15, 2019).

Fujifilm Diosynth Biotechnologies (2019). FUJIFILM diosynth biotechnologies announces $55 million USD investment to expand gene therapy development capabilities. https://fujifilmdiosynth.com/resource/fujifilm-diosynth-biotechnologies-announces-55-million-usd-investment-to-expand-gene-therapy-development-capabilities/ (last accessed November 16, 2019).

bluebird bio (2019). European medicines agency approves refined commercial manufacturing specifications for ZYNTEGLO™. http://investor.bluebirdbio.com/news-releases/news-release-details/european-medicines-agency-approves-refined-commercial (last accessed November 14, 2019).