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References
1.AveXis . AveXis announces New England Journal of Medicine publication of Phase 1 data of AVXS-101 gene replacement therapy in spinal muscular atrophy Type 1 . http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2313601 (last accessed November 17 , 2017 ).
2.
Mendell
JR
,
Al-Zaidy
S
,
Shell
R
, et al. Single-dose gene-replacement therapy for spinal muscular atrophy . New Engl J Med , 2017 ; 377 :1713 –1722 .
3.
Lewis
R.
Gene therapy replaces boy's entire epidermis . https://www.medscape.com/viewarticle/888245 - vp_1 (last accessed November 17 , 2017 ).
4.
Mavilio
F
,
Pellegrini
G
,
Ferrari
S
, et al. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells . Nat Med , 2006 ; 12 :1397 .
5.
Hirsch
T
,
Rothoeft
T
,
Teig
N
, et al. Regeneration of the entire human epidermis using transgenic stem cells . Nature , 2017 ; 551 :327 –332 .
6.
Aragona
M
,
Blanpain
C
. Commentary: transgenic stem cells replace skin . Nature , 2017 ; 551 :306 –307 .
7.bluebird bio . bluebird bio announces publication of interim data from Starbeam Study of Lenti-D™ drug product in patients with cerebral adrenoleukodystrophy (CALD) in the New England Journal of Medicine . http://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-publication-interim-data-starbeam-study (last accessed November 17 , 2017 ).
8.Spark Therapeutics . FDA advisory committee unanimously recommends approval of investigational Luxturna™ (voretigene neparvovec) for patients with biallelic RPE65-mediated inherited retinal disease . http://ir.sparktx.com/news-releases/news-release-details/fda-advisory-committee-unanimously-recommends-approval (last accessed November 11 , 2017 ).
9.
Russell
S
,
Bennett
J
,
Wellman
JA
, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, Phase 3 trial . Lancet , 2017 ; 390 :849 –860 .
10.Novartis . Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice . https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019 (last accessed November 15 , 2017 ).
11.Gilead Sciences . Kite's Yescarta™ (axicabtagene ciloleucel) becomes first CAR T therapy approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy . www.gilead.com/news/press-releases/2017/10/kites-yescarta-axicabtagene-ciloleucel-becomes-first-car-t-therapy-approved-by-the-fda-for-the-treatment-of-adult-patients-with-relapsed-or-refractory-large-bcell-lymphoma-after-two-or-more-lines-of-systemic-therapy (last accessed November 15 , 2017 ).
12.uniQure . uniQure announces hemophilia B gene therapy program to enter pivotal study with FIX-Padua cariant in 2018 . https://tools.eurolandir.com/tools/Pressreleases/GetPressRelease/?ID=3388861&lang=en-GB&companycode=nl-qure&v (last accessed November 16 , 2017 ).
13.National Institute for Health and Care Excellence . Evaluation consultation document Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency . https://www.nice.org.uk/guidance/GID-HST10005/documents/evaluation-consultation-document (last accessed November 15 , 2017 ).
14.
Miller
S
. 2017 . Gene therapy holds great promise, but big price . http://lab.express-scripts.com/lab/insights/drug-options/gene-therapy-holds-great-promise-but-big-price (last accessed November 11 , 2017 ).
15.
Kolata
G
. 2017 . New gene-therapy treatments will carry whopping price tags . The New York Times , September 11 , 2017 . https://www.nytimes.com/2017/09/11/health/cost-gene-therapy-drugs.html (last accessed November 11 , 2017 ).
16.
Montazerhodjat
V
,
Weinstock
DM
,
Lo
AW
. Buying cures versus renting health: financing health care with consumer loans . Sci Transl Med , 2016 ; 8 :327ps326 .
17.StreetInsider . Express Scripts (ESRX) CMO issues blog on CAR-Ts and gene therapies . https://www.streetinsider.com/Analyst+Comments/Express+Scripts+%28ESRX%29+CMO+Issues+Blog+on+CAR-Ts+and+Gene+Therapies/13318181.html (last accessed November 11 , 2017 ).
18.Ultragenyx Pharmaceutical . Ultragenyx completes tender offer and acquisition of Dimension Therapeutics . http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1047500 (last accessed November 13 , 2017 ).
19.REGENXBIO . Regenxbio responds to notification of a superior proposal under its merger agreement with Dimension Therapeutics . http://globenewswire.com/news-release/2017/10/02/1138738/0/en/REGENXBIO-Responds-to-Notification-of-a-Superior-Proposal-Under-Its-Merger-Agreement-With-Dimension-Therapeutics.html?f=22&fvtc=7 (last accessed November 13 , 2017 ).
20.Voyager Therapeutics . Voyager Therapeutics announces update to Parkinson's disease program . http://ir.voyagertherapeutics.com/phoenix.zhtml?c=254026&p=irol-newsArticle&ID=2312537 (last accessed November 11 , 2017 ).
21.Voyager Therapeutics . Voyager Therapeutics announces positive results from ongoing Phase 1b trial of VY-AADC01 for advanced Parkinson's disease . http://ir.voyagertherapeutics.com/phoenix.zhtml?c=254026&p=irol-newsArticle&ID=2298649 (last accessed November 11 , 2017 ).
22.Voyager Therapeutics . Voyager Therapeutics reports third quarter 2017 financial results and corporate highlights . http://ir.voyagertherapeutics.com/phoenix.zhtml?c=254026&p=irol-newsArticle&ID=2313987 (last accessed November 11 , 2017 ).
23.Gilead Sciences . Gilead Sciences to acquire Kite Pharma for $11.9 billion . www.gilead.com/news/press-releases/2017/8/gilead-sciences-to-acquire-kite-pharma-for-119-billion (last accessed November 13 , 2017 ).
