Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment
Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm comparable in potential impact on human health to...
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| Format: | Article |
| Language: | English |
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MDPI AG
2014-03-01
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| Series: | Biomedicines |
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| Online Access: | http://www.mdpi.com/2227-9059/2/1/80 |
| _version_ | 1831667708304818176 |
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| author | J. Fraser Wright |
| author_facet | J. Fraser Wright |
| author_sort | J. Fraser Wright |
| collection | DOAJ |
| description | Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm comparable in potential impact on human health to that achieved by recombinant proteins and vaccines. A challenge for the current pipeline of AAV-based investigational products as they advance through clinical development is the identification, characterization and lot-to-lot control of the process- and product-related impurities present in even highly purified preparations. Especially challenging are AAV vector product-related impurities that closely resemble the vector itself and are, in some cases, without clear precedent in established biotherapeutic products. The determination of acceptable levels of these impurities in vectors prepared for human clinical product development, with the goal of new product licensure, requires careful risk and feasibility assessment. This review focuses primarily on the AAV product-related impurities that have been described in vectors prepared for clinical development. |
| first_indexed | 2024-12-19T20:16:31Z |
| format | Article |
| id | doaj.art-425978c9b3034b51bae053c053ff48f9 |
| institution | Directory Open Access Journal |
| issn | 2227-9059 |
| language | English |
| last_indexed | 2024-12-19T20:16:31Z |
| publishDate | 2014-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Biomedicines |
| spelling | doaj.art-425978c9b3034b51bae053c053ff48f92022-12-21T20:07:09ZengMDPI AGBiomedicines2227-90592014-03-0121809710.3390/biomedicines2010080biomedicines2010080Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk AssessmentJ. Fraser Wright0Center for Cellular and Molecular Therapeutics, the Children's Hospital of Philadelphia, ARC1216C, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USAAdeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm comparable in potential impact on human health to that achieved by recombinant proteins and vaccines. A challenge for the current pipeline of AAV-based investigational products as they advance through clinical development is the identification, characterization and lot-to-lot control of the process- and product-related impurities present in even highly purified preparations. Especially challenging are AAV vector product-related impurities that closely resemble the vector itself and are, in some cases, without clear precedent in established biotherapeutic products. The determination of acceptable levels of these impurities in vectors prepared for human clinical product development, with the goal of new product licensure, requires careful risk and feasibility assessment. This review focuses primarily on the AAV product-related impurities that have been described in vectors prepared for clinical development.http://www.mdpi.com/2227-9059/2/1/80AAV vectorsimpuritiesclinical trials |
| spellingShingle | J. Fraser Wright Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment Biomedicines AAV vectors impurities clinical trials |
| title | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment |
| title_full | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment |
| title_fullStr | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment |
| title_full_unstemmed | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment |
| title_short | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment |
| title_sort | product related impurities in clinical grade recombinant aav vectors characterization and risk assessment |
| topic | AAV vectors impurities clinical trials |
| url | http://www.mdpi.com/2227-9059/2/1/80 |
| work_keys_str_mv | AT jfraserwright productrelatedimpuritiesinclinicalgraderecombinantaavvectorscharacterizationandriskassessment |