Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience-Reference-Cited by-同舟云学术

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Published:2023-03-01 Issue:5-6 Volume:34 Page:217-227
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Lomash Richa Madan¹^ORCID,Shchelochkov Oleg²,Chandler Randy J.²,Venditti Charles P.²^ORCID,Pariser Anne R.³,Ottinger Elizabeth A.¹,Gilberto V. Averion,Krishna Balakrishnan,Carsten G. Bönnemann,Philip J. Brooks,Steven J. Burden,Eggerton Campbell,Catherine Chen,Eun-Young Choi,Claire Driscoll,Oksana Dukhanina,Susan Ferry,A. Reghan Foley,Janelle Geist Hauserman,Lina Li,Donald C. Lo,Venkata Mangalampalli,Irini Manoli,Christopher Mendoza,Julien Oury,Forbes D. Porter,Deanna Portero,Lili Portilla,Jachinta Rooney,Dimah Saade,Jennifer L. Sloan,Mitali Tambe,Pramod Terse,Joshua Todd,London Toney,Carol Van Ryzin,Rodica Stan,Sury Vepa,Erik Wagner,Amy Wang,Xin Xu,Yaqun Zou,

Affiliation:

1. Therapeutic Development Branch, Division of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), NIH, Rockville, Maryland, USA

2. Organic Acid Research Section, Molecular Medicine Branch, National Human Genome Research Institute (NHGRI), NIH, Bethesda, Maryland, USA

3. Division of Rare Diseases Research Innovation, NCATS, NIH, Rockville, Maryland, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2022.232

Reference25 articles.

1. The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

2. How many rare diseases are there?

3. What Is the Purpose of the Orphan Drug Act?

4. Incentives for orphan drug research and development in the United States

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