Development of gene therapies—lessons from nusinersen

ABSTRACT The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical

trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the Food and Drug Administration (FDA), and use of

FDA’s expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases. Access through your institution Buy

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ANALYSIS AND COMPARATIVE EVALUATION OF EXPEDITED PROGRAMS FOR GENE THERAPY PRODUCTS: INSIGHTS FROM THE UNITED STATES, THE EUROPEAN UNION, JAPAN, AND SOUTH KOREA Article 10 January 2024

APPROVAL AND THERAPEUTIC VALUE OF GENE THERAPIES IN THE US AND EUROPE Article 08 November 2023 ETHICAL CHALLENGES FOR A NEW GENERATION OF EARLY-PHASE PEDIATRIC GENE THERAPY TRIALS Article 07

July 2021 REFERENCES * Nusinersen (SPINRAZA) FDA reviews and label. 2016. Available at:http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/209531Orig1s000TOC.cfm. * FDA Draft Guidance

for Industry: Target Product Profile - A Strategic Development Process Tool. 2007. Available at:www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm080593.pdf. *

FDA Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics. 2014. Available

at:http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf. Download references AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Office of Tissues and

Advanced Therapies, Center for Biologics Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA L Xu, I Irony & W W Bryan * Division of Neurology

Products, Office of New Drugs, Center for Drug Evaluation and Research, United States Food and Drug Administration, Silver Spring, MD, USA B Dunn Authors * L Xu View author publications You

can also search for this author inPubMed Google Scholar * I Irony View author publications You can also search for this author inPubMed Google Scholar * W W Bryan View author publications

You can also search for this author inPubMed Google Scholar * B Dunn View author publications You can also search for this author inPubMed Google Scholar CORRESPONDING AUTHOR Correspondence

to W W Bryan. ETHICS DECLARATIONS COMPETING INTERESTS The authors declare no conflict of interest. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Xu,

L., Irony, I., Bryan, W. _et al._ Development of gene therapies—lessons from nusinersen. _Gene Ther_ 24, 527–528 (2017). https://doi.org/10.1038/gt.2017.64 Download citation * Received: 13

March 2017 * Revised: 13 July 2017 * Accepted: 18 July 2017 * Published: 24 July 2017 * Issue Date: September 2017 * DOI: https://doi.org/10.1038/gt.2017.64 SHARE THIS ARTICLE Anyone you

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