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The state of technological advancement to address challenges in the manufacture of rAAV gene therapies.
Destro, Francesco; Wu, Weida; Srinivasan, Prasanna; Joseph, John; Bal, Vivekananda; Neufeld, Caleb; Wolfrum, Jacqueline M; Manalis, Scott R; Sinskey, Anthony J; Springs, Stacy L; Barone, Paul W; Braatz, Richard D.
Afiliação
  • Destro F; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Wu W; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Srinivasan P; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Joseph J; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Bal V; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Neufeld C; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Wolfrum JM; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Manalis SR; Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Mechanical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Sinskey AJ; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA, USA.
  • Springs SL; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA. Electronic address: ssprings@mit.edu.
  • Barone PW; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA. Electronic address: pbarone@mit.edu.
  • Braatz RD; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, MA, USA. Electronic address: braatz@mit.edu.
Biotechnol Adv ; 76: 108433, 2024 Nov.
Article em En | MEDLINE | ID: mdl-39168354
ABSTRACT
Current processes for the production of recombinant adeno-associated virus (rAAV) are inadequate to meet the surging demand for rAAV-based gene therapies. This article reviews recent advances that hold the potential to address current limitations in rAAV manufacturing. A multidisciplinary perspective on technological progress in rAAV production is presented, underscoring the necessity to move beyond incremental refinements and adopt a holistic strategy to address existing challenges. Since several recent reviews have thoroughly covered advancements in upstream technology, this article provides only a concise overview of these developments before moving to pivotal areas of rAAV manufacturing not well covered in other reviews, including analytical technologies for rapid and high-throughput measurement of rAAV quality attributes, mathematical modeling for platform and process optimization, and downstream approaches to maximize efficiency and rAAV yield. Novel technologies that have the potential to address the current gaps in rAAV manufacturing are highlighted. Implementation challenges and future research directions are critically discussed.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos Limite: Animals / Humans Idioma: En Revista: Biotechnol Adv Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Dependovirus / Vetores Genéticos Limite: Animals / Humans Idioma: En Revista: Biotechnol Adv Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos