Gene Therapy Briefs

Hinderer C , Katz N , Buza EL , et al. Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN. Hum Gene Ther, 2018 Feb 12 [Epub ahead of print]; DOI:10.1089/hum.2018.015.

Flotte T , Büning H. Severe toxicity in nonhuman primates and piglets with systemic high-dose administration of adeno-associated virus serotype 9–like vectors: putting patients first. Hum Gene Ther, 2018 Feb 7 [Epub ahead of print]; DOI:10.1089/hum.2018.021.

Philippidis A. Virus supply vexes gene therapy developers, CMOs. Genetic Engineering & Biotechnology News, December 14, 2017. https://www.genengnews.com/gen-exclusives/virus-supply-vexes-gene-therapy-developers-cmos/77901021/ (last accessed February 26, 2018).

Oxford BioMedica. Oxford BioMedica announces collaboration and licence agreement with Bioverativ in the field of haemophilia gene therapy. http://www.oxfordbiomedica.co.uk/news-media/press-release/oxford-biomedica-announces-collaboration-and-licence-agreement-bioverativ (last accessed February 26, 2018).

Lonza. Lonza to establish cell- and gene-therapy centers of excellence to accelerate growth. https://www.lonza.com/about-lonza/media-center/news/Tensid/2018-02-14-06-00-English.aspx (last accessed February 28, 2018).

Cell and Gene Therapy Catapult. CGT Catapult announces viral vector manufacturing technology collaboration with Freeline Therapeutics. https://ct.catapult.org.uk/news-media/manufacturing-news/viral-vector-manufacturing-freeline (last accessed February 26, 2018).

Innovate UK. Results of competition: Health & Life Sciences—Round 2—12–24 Months. https://www.gov.uk/government/uploads/system/uploads/attachment_data/file/637043/Health___Life_Sciences_-_Round_2_-_12-24_Months_-_Competition_Results.pdf (last accessed February 26, 2018).

REGENXBIO. REGENXBIO enhances gene therapy manufacturing capabilities by entering into strategic partnership with FUJIFILM Diosynth Biotechnologies. http://ir.regenxbio.com/phoenix.zhtml?c=254175&p=RssLanding&cat=news&id=2328004 (last accessed February 27, 2018).

Novartis. Novartis exclusively licenses first ophthalmology gene therapy in all markets outside the US, a milestone for patients with rare inherited vision loss. https://www.novartis.com/news/media-releases/novartis-exclusively-licenses-first-ophthalmology-gene-therapy-all-markets-outside-us-milestone-patients-rare-inherited-vision-loss (last accessed February 27, 2018).

Spark Therapeutics. Spark Therapeutics enters into a licensing and supply agreement for investigational voretigene neparvovec outside the U.S. http://ir.sparktx.com/news-releases/news-release-details/spark-therapeutics-enters-licensing-and-supply-agreement (last accessed February 27, 2018).

Celgene and Juno Therapeutics. Celgene Corporation to acquire Juno Therapeutics, Inc., advancing global leadership in cellular immunotherapy. http://ir.celgene.com/releasedetail.cfm?releaseid=1054833 (last accessed February 24, 2018).

Sangamo Therapeutics and Pfizer. Sangamo and Pfizer announce collaboration for development of zinc finger protein gene therapy for ALS. https://www.pfizer.com/news/press-release/press-release-detail/sangamo_and_pfizer_announce_collaboration_for_development_of_zinc_finger_protein_gene_therapy_for_als (last accessed February 24, 2018).

AbbVie and Voyager Therapeutics. AbbVie and Voyager Therapeutics announce global strategic collaboration to develop potential new treatments for Alzheimer's disease and other tau-related neurodegenerative diseases. https://news.abbvie.com/news/abbvie-and-voyager-therapeutics-announce-global-strategic-collaboration-to-develop-potential-new-treatments-for-alzheimers-disease-and-other-tau-related-neurodegenerative-diseases.htm (last accessed February 27, 2018).

U.S. Securities and Exchange Commission. Form 8-K of Voyager Therapeutics, filed February 20. https://www.sec.gov/Archives/edgar/data/1640266/000110465918010520/a18-6349_18k.htm (last accessed February 27, 2018).