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Current state of U.S. Food and Drug Administration regulation for cellular and gene therapy products: potential cures on the horizon.

Abstract：

:Cellular & Gene Therapies (CGTs) are complex products, which have been key foci of the International Society for Cell & Gene Therapy (ISCT). For this ISCT North American Legal & Regulatory Affairs Committee review publication, CGTs include but are not limited to somatic cell-based therapies, pluripotent cell-derived cell-based therapies, gene- or non-gene-modified or gene edited versions of these cell-based therapies, in vivo gene therapies, organ/tissue engineered products, and relevant combination products. These products are regulated by the Food and Drug Administration (FDA) in the United States. This publication reviews selected laws, regulations, guidance, definitions, processes, types of meetings and submissions, and other key factors that the FDA follows and implements to regulate and support development of these types of products. These factors may be considered in order to help current and potential product developers/sponsors/applicants navigate through FDA regulatory pathways. We also review expedited programs including types of Designations available at the FDA, and their specific eligibility criteria. We include FDA and other stakeholder resources to consider regarding CGT regulation, to help prepare for CGT development and subsequent FDA approval.

journal_name

Cytotherapy

journal_title

Cytotherapy

authors

Mendicino M,Fan Y,Griffin D,Gunter KC,Nichols K

doi

10.1016/j.jcyt.2019.04.002

subject

Has Abstract

pub_date

2019-07-01 00:00:00

pages

699-724

issue

7

eissn

1465-3249

issn

1477-2566

pii

S1465-3249(19)30387-1

journal_volume

21

pub_type

杂志文章,评审

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