Sartorius - November 2021 - Simplifying Adeno Associated Virus - 3

Best Practice for
rAAV Vector Production in
Suspension Cell Culture
Recombinant Adeno-associated virus
(rAAV) has been studied for many decades
and is now the viral vector of choice for
a range of ground-breaking research
and clinical applications. In recent years,
rAAV has come to the fore for use in gene
therapy to treat rare diseases with a strong
pipeline of clinical candidates and two
therapies approved by the FDA: Luxturna®
for the treatment of Leber's congenital
amaurosis and Zolgensma®
for spinal
muscular atrophy. The success of rAAV
for use in rare disease gene therapies has
sparked pre-clinical and clinical studies
in its wider use, notably to treat a range of
cancers¹ which is boosting the need for
rAAV manufacturing globally.
Amélie Boulais
Head of Market Entry Strategy
Viral based Therapeutics, Sartorius
amelie.boulais@sartorius.com
So why is rAAV such a popular viral vector?
Cécile Robin, Process Operation Manager
for the French " industrial integrator " CPV
at INSERM UMR 1089 at the University of
Nantes states: " rAAV has been developed
for clinical use for over 20 years now, so
it has a good safety and efficacy record. "
This alone makes it a good choice where
it is going to be given directly to a patient.
Véronique Blouin-Tavel, PhD, Vector Core
and Process Development Manager at the
University of Nantes adds: " rAAV allows
long-term transgene expression with a
lower immunogenicity unlike other types
of gene delivery mechanisms. "
3 | GENengnews.com
The appeal of rAAV for gene therapy
applications lies in its inability to integrate
into the genome and its ability to
transduce dividing and non-dividing
cells. Additionally, rAAV has multiple
serotypes that can target different cells,
tissues, and organs which means it can
be designed and optimized to target
very specific cells which is a significant
advantage for gene therapy applications.
Due to its versatility and safety, rAAV has
become a sought-after platform by many
organizations to engineer and deliver
effective gene therapy mechanisms for a
wide range of clinical applications.
This has led to a dramatic increase in
gene therapy development in recent
years, which has resulted in a global
shortage of rAAV manufacturing
capacity². Recently, at some Contract
Development and Manufacturing
Organizations (CDMOs) those gene
therapy demands are now competing
for manufacturing resources with
COVID-19 vaccines which is impacting
near term capacity and timelines². Robin
comments: " Since we are a pioneering
lab that is experienced with rAAV vector
production, our services are now in
greater demand, and we are working with
partners to produce more research and
pre-clinical grade rAAV at up to
50 L scale. "
https://www.genengnews.com/

Sartorius - November 2021 - Simplifying Adeno Associated Virus

Table of Contents for the Digital Edition of Sartorius - November 2021 - Simplifying Adeno Associated Virus

Sartorius - November 2021 - Simplifying Adeno Associated Virus - 1
Sartorius - November 2021 - Simplifying Adeno Associated Virus - 2
Sartorius - November 2021 - Simplifying Adeno Associated Virus - 3
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