Application to AAV Manufacturing
Automatic bioprocess can be achieved to link
host cell culture, viral packaging and viral purification
in AAV manufacturing because the new technology
resolves the problems of AAV purification. This
bioprocess will be perfect for AAV manufacturing for
clinic trials and new medicine production, which
features ultrapurity, high recovery, time-saving and
no concern of cytotoxicity.
AAV is the Safety Vector for Gene Therapy
Biological safety is the first concern in use for gene therapy. Through 30 years of clinic
trials, three of
in vivo gene therapies were approved, including Glybera by European
Medicines Agency in 2012, Luxturna by FDA in 2017 and Zolgensma by FDA in 2019.
The viral vector of AAV is used to deliver the target genes in the three gene therapies,
indicating that AAV as a safety vector for clinic use is officially approved. AAV is a member
of parvovirus family, studied for 50 years. As a viral vector, AAV technology has been
extensively developed, including convenient viral cloning, packaging full functional
virions, expressing term and serotype specificity. Indeed, this small viral vector shows
many advantages in both basic research and clinic application over other viral vectors.
AAV Manufacturing: a Limit in Gene Therapy
Technology for AAV purification as technical support is missing but needed in gene
therapy research & development to address many issues including construction of AAV vector
and screening and testing recombinant AAV (rAAV). Therefore, AAV manufacturing becomes
an obstruction in gene therapy. For high quality and large amount of AAV preparation that is
required to ensure the experimental accuracy and meet the need in R&D and clinic use as well,
thus promoting the R&D progress, a high efficient technology for AAV purification is expected
to resolve the issues in AAV purification. When the difficulties of AAV purification are
overcome, the cost of gene therapy will be reduced and, as a consequence, its pricing will be
moderated close to patients affordability from the overpriced. However, there are two major
difficulties (as shown below) insurmountable in exploring a new, efficient technology for AAV
Our New Technology Makes AAV in High Quality
and Large Amount
The new technology is a creative method employing a new found principle that highly
efficiently surmounts the two difficulties in AAV purification motioned above and produces
high quality AAV regardless the volume of preparation. It includes only three major steps, cell
lysis, fractionation and a chromatography column, meeting all needs in amount of AAV
prepared for gene therapy. Its significances of importance for gene therapy include the main
aspects as described in the table below.
Significances of importance
High quality virus ensure accuracy and repeatability
Small preps meet the need of innovation and enhance the efficiency
Large scaled manufacturing meets the need of clinic trials and production
Fast virus preparation meets all needs in R&D, promoting gene therapy process
Resolve issues of AAV purification leading to lower cost and moderately pricing
Bioprocess of the New Tech
The company
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