MurikinatiS.Gene Therapy: Development, Investment and Regulatory Insights. 2023. Available from: https://www.criver.com/eureka/gene-therapy-development-investment-and-regulatory-insights [Last accessed: August27, 2023].
3.
BarrettD, WendlandA, RoseD, et al. Gene, Cell, + RNA Therapy Landscape Report Q3 2023 Quarterly Data Report. American Society of Gene & Cell Therapy-Waukesha, Wisconsin; 2023.
4.
United States Food & Drug Administration. Current FDA Approved Gene and Cell Therapies. US Food & Drug Administration, Silver Spring, Maryland; 2023. Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products [Last accessed: August31, 2023].
5.
United States Food & DrugAdministration. Statement from FDA Commissioner Scott GottliebM.D. and Peter MarksM.D., Ph.D., Director of the Center for Biologics Evaluation and Research on New Policies to Advance Development of Safe and Effective Cell and GeneTherapies. 2019.
6.
WongCH, LiD, WangN, et al. Estimating the Financial Impact of Gene Therapy in the U.S. Cambridge, MA; 2021.; doi: 10.3386/w28628
7.
United States Food & Drug Administration. Establishment of the Office of Therapeutic Products. Food & Drug Administration, Silver Spring, Maryland; 2023. Available from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/establishment-office-therapeutic-products [Last accessed: November7, 2023].
8.
U.S. Food & Drug Administration. FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies. Food & Drug Administration, Silver Spring, Maryland; 2023. Available from: https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies [Last accessed: October17, 2023].
9.
MarsdenG, TowseA, PearsonSD, et al. Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value. Institute for Clinical and Economic Review, Boston, Massachusetts; 2017.
10.
TolbertJ, DrakeP, DamicoA. Key Facts about the Uninsured Population. KFF, 2022.
11.
CollinsSR, BhupalHK, DotyMM. Health Insurance Coverage Eight Years after the ACA: Fewer Uninsured Americans and Shorter Coverage Gaps, but More Underinsured. The Commonwealth Fund, 2019.
12.
DeMartinoP, HaagMB, HershAR, et al.A budget impact analysis of gene therapy for sickle cell disease. JAMA Pediatr, 2021; 175(6):617; doi: 10.1001/jamapediatrics.2020.7140
13.
DunnA.Kids with “bubble Boy” Disease Are Dying—Even Though Drug Companies Have Found a Cure. 2022. Available from: https://www.businessinsider.com/bubble-boy-disease-cure-but-drug-companies-wont-provide-it-2022-7 [Last accessed: August27, 2023].
14.
PagliaruloN. As BiotechRetreats, Gene Therapy Companies Retrench and RedrawPlans. 2022. Available from: https://www.biopharmadive.com/news/gene-therapy-biotech-restructuring-jobs-pipeline/620980 [Last accessed: August 28, 2023].2
15.
FidlerB.‘The Music Stopped’: Biotech Rout Leaves Drug Startups Grounded as Demand Slumps for IPOs. 2022. Available from: https://www.biopharmadive.com/news/biotech-ipo-venture-startup-investors-market-downturn/618205 [Last accessed: August27, 2023].
16.
OffitPA.Why are pharmaceutical companies gradually abandoning vaccines?. Health Aff, 2005; 24(3):622–630; doi: 10.1377/hlthaff.24.3.622
17.
DouglasRG, SamantVB. The vaccine industry. In: Plotkin's Vaccines. Elsevier; 2018; pp. 41–50.e1; doi: 10.1016/B978-0-323-35761-6.00004-3
18.
WilsonJ, MangalA. Weathering the Storm: Cell and Gene's Economic Downturn. 2023. Available from: https://themedicinemaker.com/business-regulation/how-to-survive-cell-and-genes-economic-downturn [Last accessed: August30, 2023].
WolfM. Dallas-Based Biotech Firm Taysha Drops Gene Therapy Following FDA Meeting. Dallas Morning News, 2023.
21.
LynchC.Taysha Drops Gene Therapy Candidate as FDA Again Calls for Study Changes. 2023. Available from: https://www.biospace.com/article/taysha-drops-gene-therapy-candidate-as-fda-again-calls-for-study-changes [Last accessed: September22, 2023].
United States Food & Drug Administration. FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy. Food & Drug Administration, Silver Spring, Maryland; 2023. Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy [Last accessed: November16, 2023].
24.
ArmstrongA.As Novartis Pulls Back from Legacy Sickle Cell Program, Intellia CEO Calls the Market “Wide Open.”. 2023. Available from: https://www.fiercebiotech.com/biotech/novartis-pulls-back-legacy-sickle-cell-program-intellia-ceo-calls-market-wide-open [Last accessed: August28, 2023].
25.
GoodwinK.Novartis Drops SCD Gene Therapy Program with Intellia. 2023. Available from: https://www.biospace.com/article/novartis-drops-scd-gene-therapy-program-with-intellia- [Last accessed: August28, 2023].
26.
JohnsonV.Graphite Bio Drops Lead Sickle Cell Gene-Edited Cell Therapy. 2023. Available from: https://www.cgtlive.com/view/graphite-bio-drops-lead-sickle-cell-gene-edited-cell-therapy [Last accessed: August28, 2023].
27.
Graphite BioInc., Graphite Bio Announces Voluntary Pause of Phase 1/2 CEDAR Study of Nulabeglogene Autogedtemcel (Nula-Cel) for Sickle Cell Disease [PressRelease}. South San Francisco, Calif., Graphite Bio, Inc.; 2023.
28.
BayerM.Sangamo Shelves Sickle Cell Therapy, Joining Rivals Forced to Rethink Development Plans. 2023. Available from: https://www.fiercebiotech.com/biotech/sangamo-shelves-sickle-cell-therapy-joining-rivals-forced-rethink-development-plans-disease [Last accessed: October12, 2023].
29.
United States Food & Drug Administration. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. US Food and Drug Administration, Silver Spring, Maryland; 2023. Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease [Last accessed: December28, 2023].
30.
M M, YoungS. UK Authorises Gene Therapy for Blood Disorders in World First. Reuters, 2023.
31.
SarpatwariA, DiBelloJ, ZakarianM, et al.Competition and price among brand-name drugs in the same class: A systematic review of the evidence. PLoS Med, 2019; 16(7):e1002872; doi: 10.1371/journal.pmed.1002872
32.
ElizabethCohen, LaurenMascarenhas. A Drug Company Abandoned a Treatment for ‘Bubble Boy Disease.’ After a 5-Year Fight, This Little Girl Is about to Get It. 2023. Available from: https://www.cnn.com/2023/04/27/health/bubble-boy-treatment-fight/index.html [Last accessed: August28, 2023].
33.
KohnDB, BoothC, ShawKL, et al.Autologous ex vivo lentiviral gene therapy for adenosine deaminase deficiency. N Engl J Med, 2021; 384(21):2002–2013; doi: 10.1056/NEJMoa2027675
34.
HirschlerB. GSK Slims Portfolio with Sale of Rare Disease Gene Therapy Drugs. Reuters, 2018.
35.
Global Outreach Committee. Goals of Global Outreach. n.d.
36.
World Health Organization. WHO Approach towards the Development of a Global Regulatory Framework 7 for Cell and Gene Therapy Products. World Health Organization, Geneva, Switzerland; 2022. Available from: https://cdn.who.int/media/docs/default-source/biologicals/bs2424_cgtp-document-for-2nd-public-consultation.pdf [Last accessed: November3, 2023].
37.
The World Bank. World Bank Open Data. n.d. Available from: https://data.worldbank.org [Last accessed: November26, 2023].
38.
GiuglianiR, Castillo TaucherS, HafezS, et al.Opportunities and challenges for newborn screening and early diagnosis of rare diseases in Latin America. Front Genet, 2022; 13; doi: 10.3389/fgene.2022.1053559
39.
Mission, Vision andObjectives.Lautaro Foundation Needs You. n.d. Available from: https://fundacionlautarotenecesita.org [Last accessed: November30, 2023].
40.
PagliaruloN.Editas to Cut Jobs, Narrow Research Focus in Restructuring. 2023. Available from: https://www.biopharmadive.com/news/editas-restructuring-layoffs-edit-301-sickle-cell/639912 [Last accessed: August27, 2023].
41.
DunleavyK.With the Pricing Situation “untenable” in Europe, Bluebird Will Wind down Its Operations in the “Broken” Market. 2021. Available from: https://www.fiercepharma.com/pharma/situation-untenable-bluebird-will-wind-down-its-operations-broken-europe [Last accessed: October15, 2023].
42.
FoxT, BuerenJ, CandottiF, et al.Access to gene therapy for rare diseases when commercialization is not fit for purpose. Nat Med, 2023; 29(3):518–519; doi: 10.1038/s41591-023-02208-8
43.
U.S. Food & Drug Administration. Rare Diseases at FDA. 2022. Available from: https://www.fda.gov/patients/rare-diseases-fda#top [Last accessed: October20, 2023].
44.
Immune Deficiency Foundation. ADA-SCID Gene Therapy Trials to Resume in 2023. Immune Deficiency Foundation, 2022.
45.
ValsecchiMC.Rescue of an orphan drug points to a new model for therapies for rare diseases. Nat Italy, 2023; doi: 10.1038/d43978-023-00145-1
46.
WhitlockJ.Families Raised Millions and Handed Rare Disease Therapies off to Biotechs. But Companies Have Backed Out. 2023. Available from: https://beyondbatten.org/wp-content/uploads/2023/06/2a8728af27bd6075f13ed0a62f66fbb4endpoints.pdf [Last accessed: August28, 2023].
Elpida TherapeuticsS. Gene Therapy Programs for Ultra-Rare Diseases to Advance with Ground-Breaking Launch of Elpida Therapeutics SPC. Everyone's Internet News Presswire, 2023.
51.
Elpida Therapeutics. Accelerating Life Saving Gene Therapies. n.d. Available from: https://www.elpidatx.com [Last accessed: November26, 2023].
52.
WhitlockJ. A Biotech Paused a Gene Therapy Hatched by Families. Now They Want Control. Endpoint News, 2022.
53.
Rare Daily Staff. Taysha Pauses Programs, Cuts Workforce to Extend Cash Runway. Global Genes, 2022.
54.
Homology MedicinesInc., Homology Medicines Announces Plan to Evaluate Strategic Options for the Company and its Genetic MedicinesPrograms, Including HMI-103 Gene Editing Candidate for PKU [Press Release]. Bedford, Mass.; 2023.
55.
PagliaruloN.Avrobio Halts Gene Therapy Research and Considers a Sale. 2023. Available from: https://www.biopharmadive.com/news/avrobio-strategic-alternatives-sale-gene-therapy-research/686643 [Last accessed: August28, 2023].
56.
Paul TaylorN.Avrobio Stops Programs, Halves Head Count and Searches for Exit. 2023. Available from: https://www.fiercebiotech.com/biotech/avrobio-stops-programs-halves-headcount-and-searches-exit [Last accessed: August28, 2023].
57.
PagliaruloN.Avrobio Stops Work on Rare Disease Gene Therapy after Unexpected Study Results. 2022. Available from: https://www.biopharmadive.com/news/avrobio-fabry-discontinue-gene-therapy-data/616594 [Last accessed: August28, 2023].
58.
Paul TaylorN. Novartis Dumps Rett Gene Therapy Caught up in Zolgensma Scandal in Cull of Pipeline Prospects. Fierce Biotech, 2021.
59.
GardnerJ.Pfizer Pares Back Early-Stage Research in Rare Disease, Cancer. 2023. Available from: https://www.biopharmadive.com/news/pfizer-early-stage-rare-disease-cancer-pare-back/639824 [Last accessed: August27, 2023].
60.
WaldronJ.Selecta Lays off 25% as Unpartnered Gene Therapies Take a Back Seat. 2023. Available from: https://www.fiercebiotech.com/biotech/selecta-lays-25-unpartnered-gene-therapies-take-back-seat [Last accessed: August28, 2023].
61.
BrysonS.Three DMD Gene Therapy Programs Terminated by Astellas, Citing Data Steve Bryson, PhD Avatar. 2022. Available from: https://musculardystrophynews.com/news/dmd-gene-therapy-programs-terminated-astellas-preclinical-data [Last accessed: August28, 2023].
62.
LiuA.Fierce Pharma Asia—Takeda's Gene Therapy Pivot and Pay-for-Delay Lawsuit; Astellas' Write-Offs. 2023. Available from: https://www.fiercepharma.com/pharma/takeda-gene-therapy-pivot-new-pay-delay-lawsuit-astellas-write-offs [Last accessed: August28, 2023].
63.
WaldronJ.Takeda Confirms Fallout from Gene Therapy R&D Refocus: Up to 186 Layoffs. 2023. Available from: https://www.fiercebiotech.com/biotech/takeda-confirms-fallout-gene-therapy-rd-refocus-186-redundancies [Last accessed: September21, 2023].
64.
SmithJ.Facing Tough Markets, Freeline Therapeutics Drops Fabry Disease Gene Therapy and Lays off 30% of Workforce. 2023. Available from: https://www.biopharma-reporter.com/Article/2023/04/06/Facing-tough-markets-Freeline-Therapeutics-drops-Fabry-disease-gene-therapy-and-lays-off-30-of-workforce [Last accessed: August28, 2023].
65.
RTTNews.com. PTC Therapeutics Discontinues Some Programs In Gene Therapy; Stock Down. 2023. Available from: https://www.nasdaq.com/articles/ptc-therapeutics-discontinues-some-programs-in-gene-therapy-stock-down [Last accessed: August28, 2023].
66.
ManalacT.ROCHE DROPS HEMOPHILIA A CANDIDATE, PREPARES FOR THE IMPACTS OF IRA. 2023. Available from: https://www.biospace.com/article/roche-drops-hemophilia-a-candidate-prepares-for-the-impacts-of-ira [Last accessed: August27, 2023].
67.
JohnsonV.No Time to Pause: Companies Abandon Lagging Cell Therapy Programs. 2023. Available from: https://www.cgtlive.com/view/no-time-to-pause-companies-abandon-lagging-cell-therapy-programs [Last accessed: August27, 2023].
68.
JensenK.Orchard Abandons Promising Gene Therapy for Rare Immune Disorder. 2021. Available from: https://www.biopharmadive.com/news/orchard-abandon-gene-therapy-ada-scid/601232 [Last accessed: August27, 2023].
69.
PagliaruloN.Orchard Turns to Layoffs in Cutting Gene Therapy Research. 2023. Available from: https://www.biopharmadive.com/news/orchard-layoffs-restructuring-gene-therapy/621261 [Last accessed: August27, 2023].
70.
CarrollJ. As Shares Tank, Orchard Once Again Restructures, Shedding Gene Therapy Programs and a Large Part of the Staff. Endpoints News, 2022.
71.
PhilippidisA. Rough Passage: Gene Therapy Developer Hunkers Down afterJob, PipelineCuts. 2023. Available from: https://www.genengnews.com/gen-edge/rough-passage-gene-therapy-developer-hunkers-down-after-job-pipeline-cuts/[Last accessed: August 27, 2023].
72.
PagliaruloN. Bluebird, Winding down in Europe, Withdraws Another Rare Disease Gene Therapy. BioPharma Dive, 2021.
GelmanM. Amicus and Perceptive Call off SPAC Merger for Gene Therapy Spinout after Recent Setback. EndPointNews, 2022.
75.
StansfieldN.Expert Insights on Gene Therapy for International Batten Awareness Day. 2023. Available from: https://www.cgtlive.com/view/expert-insights-gene-therapy-international-batten-awareness-day [Last accessed: October12, 2023].
76.
ArmstrongA. JPM 2022: Amicus Axes Gene Therapy Program for Type of Batten Disease, Advances Another. Fierce Biotech, 2022.
77.
JensenK.A Once Buzzy Gene Therapy Startup Lays off Staff and Shuts Down. 2023. Available from: https://www.biopharmadive.com/news/intergalactic-therapeutics-layoffs-shut-down-gene-therapy-startups/689676 [Last accessed: August27, 2023].