Apr
29
2020
On demand

Advances in gene therapy delivery: next-generation transfection reagent for large-scale AAV manufacturing

The number of ATMP therapeutic-based medicines for inherited genetic disorders is in constant growth, with a global 32% increase in new clinical trials in the last 4 years. ATMPs have demonstrated their success with already more than ten approved for commercialization.

The success of AAV as the most promising viral vector for gene therapy is due to low immunogenicity, broad tropism and non-integrating properties. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV. Transient transfection of suspension cells is the most commonly used production platform, as it offers significant flexibility for cell and gene therapy development. However, this method shows some limitations in large scale bioreactors: inadequate transfection protocol, reduced transfection efficiency and lower productivity.

To address this concern, we present data on the novel transfection reagent showing:

i) increased AAV titers
ii) improved transfection protocol for large scale bioreactors
iii) reproducibility of viral titers at different production scale

The aforementioned optimized parameters make this novel transfection reagent ideal for cell and gene therapy developers by combining the flexibility of transient transfection with scalability and speed to market.

Attendees learn:

  • How to improve AAV viral vector yields by focusing on transfection step
  • How to perform transfection at large scale
  • How to ensure reproducibility at different production scales

Mathieu Porte
BioProduction R&D Manager, Polyplus-transfection
Malik Hellal
Senior Scientist, Chemistry at Polyplus-transfection

SPEAKERS

Mathieu Porte
BioProduction R&D Manager, Polyplus-transfection
Malik Hellal
Senior Scientist, Chemistry at Polyplus-transfection

You might also like

Thinking translational: Things to consider in CAR T cell research workflows

S Rösch, PhD
Saskia Rösch, PhD
Saskia Rösch, PhD
Global Product Manager at Miltenyi Biotec
22 Jun 2022
36
Days
8
Hrs
35
Min
Register

Accelerating cell therapy discovery & development with non-viral gene...

D Hermanson
X Patrinostro
David Hermanson
David Hermanson
Senior Manager of R&D Applications, Cell and Gene Therapy at Bio-techne
Xiaobai Patrinostro
Xiaobai Patrinostro
Manager of Genome Engineering Services, Cell and Gene Therapy at Bio-techne
16 Jun 2022
29
Days
16
Hrs
35
Min
Register

Applying an HPLC analytical platform for mRNA process monitoring

N Pavlin
Nejc Pavlin
Nejc Pavlin
Project Manager in Process Analytics Development at BIA Separations
7 Jun 2022
21
Days
8
Hrs
35
Min
Register

Optimizing E. Coli cell growth performance with in-line, real-time OD600...

T Buch
R Shanbaky
Tanja Buch
Tanja Buch
Technical Director at Biifactory Competence Center
Ramsey  Shanbaky
Ramsey Shanbaky
Bioprocess Applications Manger at C Technologies, Inc - a Repligen Company
1 Jun 2022
14
Days
16
Hrs
35
Min
Register

Monitoring of circulating CAR T cells

A Graband,
D Hünecke,
D Bremm
Annika Graband
Annika Graband
Product Manager, Cell Analysis Reagents at Miltenyi Biotec
Dr. Sabine Hünecke
Dr. Sabine Hünecke
Head of Laboratory, Stem Cell Transplantation and Immunotherapy at University Hospital Frankfurt
Dr. Melanie Bremm
Dr. Melanie Bremm
Deputy Head of Laboratory, Stem Cell Transplantation and Immunotherapy at University Hospital Frankfurt
31 May 2022
14
Days
8
Hrs
35
Min
Register