Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies

Autor: Jacqueline Wolfrum, Scott R. Manalis, Andrew J. Maloney, Georgios Katsikis, Moo Sun Hong, Anthony J. Sinskey, Sha Sha, Richard D. Braatz, Andreas L. Gimpel, Stacy L. Springs, Tam Nguyen, Paul W. Barone

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 740-754 (2021)
Molecular Therapy-Methods & Clinical Development, 20
Molecular Therapy. Methods & Clinical Development

The optimization of upstream and downstream processes for production of recombinant adeno-associated virus (rAAV) with consistent quality depends on the ability to rapidly characterize critical quality attributes (CQAs). In the context of rAAV produc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3ce723cc3f717edcd95d881946858618
http://www.sciencedirect.com/science/article/pii/S2329050121000255

Macroscopic modeling of bioreactors for recombinant protein producing Pichia pastoris in defined medium

Autor: Moo Sun Hong, Andrew J. Maloney, Tarit Mukhopadhyay, J. Christopher Love, Andrew M. Biedermann, Richard D. Braatz, M. Lourdes Velez-Suberbie, Kerry R. Love

Publikováno v: Biotechnology and Bioengineering. 118:1199-1212

The methylotrophic yeast Pichia pastoris is widely used as a microbial host for recombinant protein production. Bioreactor models for P. pastoris can inform understanding of cellular metabolism and can be used to optimize bioreactor operation. This a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2e9d460d2b88ecabfab127ec87e1e77d
https://doi.org/10.1002/bit.27643

A Virtual Plant for Integrated Continuous Manufacturing of a Carfilzomib Drug Substance Intermediate, Part 3: Manganese-Catalyzed Asymmetric Epoxidation, Crystallization, and Filtration

Autor: Lauren R. Graham, Rahul P. Sangodkar, Xiaoxiang Zhu, Elçin Içten, Gerard Capellades, Andrew J. Maloney, Richard D. Braatz, Daniel J. Griffin, Ayman Allian, Seth Huggins, Roger Hart, Matthew G. Beaver, Pablo Rolandi, Derek B. Brown, Shawn D. Walker

Publikováno v: Organic Process Research & Development. 24:1891-1908

This article describes the process characterization and development of models to inform a process control strategy to prepare (R,R)-epoxy ketone 2, an intermediate in the manufacture of carfilzomib...

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::40548ea997dd0c944901fd9c2ca06b46
https://doi.org/10.1021/acs.oprd.0c00189

Mechanistic Model for Production of Recombinant Adeno-associated Virus via Triple Transfection of HEK293 Cells

Autor: Moo Sun Hong, Andrew J. Maloney, Paul W. Barone, Caleb Neufeld, Tam Nguyen, Richard D. Braatz, Sha Sha, Anthony J. Sinskey, Stacy L. Springs, Jacqueline Wolfrum

Publikováno v: Elsevier
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 642-655 (2021)

Manufacturing of recombinant adeno-associated virus (rAAV) viral vectors remains challenging, with low yields and low full:empty capsid ratios in the harvest. To elucidate the dynamics of recombinant viral production, we develop a mechanistic model f

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::add1e3191638df02336f602530916028
https://hdl.handle.net/1721.1/135559

Green Polymer Chemistry: Biobased Materials and Biocatalysis

Autor: H. N. Cheng, Richard A. Gross, Patrick B. Smith, Judit E. Puskas, Marcela Castano, Attila L. Gergely, Karla A. Barrera-Rivera, Ángel Marcos-Fernández, Antonio Martínez Richa, Florian Pion, Armando Félix Reano, Mouandhoime Zahahe Oulame, Imane Barbara, Amandine Léa Flourat, Paul-Henri Ducrot, Florent Allais, Andrew J. Maloney, Chenbo Dong, Alan S. Campbell, Cerasela Zoica Dinu, Jun-ichi Kadokawa, Gregory L. Côté, Christopher D. Skory, John Masani Nduko, Jian Sun, Seiichi Taguchi, Charles Romain, Charlotte K. Williams, J. P. MacDonald, M. P. Shaver, Seiichi Tada, Takanori Uzawa, Yoshihiro Ito, Nacú Hernández, Mengguo Yan, R. Christopher Williams, Eric Cochran, Kathleen O’Leary Havelka, Gregory E. Gerhardt, Jian Hong, Djavan Hairabedian, Zoran S Petrović, Andrew Myers, Atanu Biswas, Vijay Mannari, Chintankumar Patel, Wenyin Li, Ali Kiamanesh, Sanghoon Kim, Jonathan Jacoby, Marie-José Stébé, Nadia Canilho, Andreea Pasc, Giada Lo Re, Philippe Dubois, Jean-Marie Raquez, Silvia D. Luebben, James W. Raebiger, Kiyohiko Toyama, Makoto Soyama, Shukichi Ta