Viralgen - How we do it

Triple-plasmid transfection

Viralgen’s manufacturing platform relies on technologies licensed from AskBio to manufacture AAV. We use a proprietary cell line, called Pro10™, derived from HEK293 cells and maintained and cultured in animal-derived component-free suspension-adapted cell culture. Pro10™ is a unique high yield universal system that can produce all serotypes and chimeric forms of rAAV.

Viralgen’s upstream process is plasmid-based triple transfection using 2L to 2000L single-use stirred-tank bioreactors. After expansion, Pro10 cells are co-transfected with (i) the Transgene plasmid, (ii) a plasmid carrying pAAVrep2capX and (iii) a helper plasmid carrying the VA-RNA, E2A and E4 helper genes of the Adenovirus serotype 5.

Viralgen has optimized RepCap and Helper plasmids available for use as a part of its platform approach to upstream manufacturing.

Pro10^TM cell line

Viralgen’s purification process ensures process residuals are minimized and prioritizes full capsids with a scalable, agnostic approach.

Material from the bioreactor is clarified by depth filtration prior to the first purification step by immunoaffinity chromatography.

The pre-purified product is then subjected to a DNA-containing AAV “full” particles enrichment step using iodixanol gradient ultracentrifugation prior to a second chromatography step using an ion exchange matrix.

A final ultrafiltration/diafiltration step is then performed by tangential flow filtration into formulation buffer prior to a terminal 0.2 µm filtration. The resulting rAAV drug substance is then stored at 2-8ºC and characterized by the Viralgen QC department to prepare for drug product filling.

The purified drug substance may then be diluted (if needed) to the target concentration with formulation buffer, filled into final vials and continues the process of characterization and release by Viralgen’s QC staff.

Business model and practices

At Viralgen, we strive to ensure that our business practices and the support we give you as a client reflect our passion for and understanding of our industry and customers. Everyone, ourselves included, has struggled at times with the complexity of decision-making and contracting for manufacturing of gene therapies – our goal is to be responsive, flexible and action-oriented to ensure the business relationship enables the work we do.

Dedicated program team

To that end, we put you at the center of our work at every opportunity. Whether it is our dedicated team of project managers, our regulatory support staff, or our commercial supply approach that reflects the potential curative nature of gene therapies, we hope you always feel the care we take in our work and the gratitude we have to be able to help bring these therapies to life.

Business principles

At Viralgen, we strive to ensure that our business practices, quality, and the support we give you as a client reflect our passion for and understanding of our industry and customers. Everyone, ourselves included, has struggled at times with the complexity of decision-making and contracting for manufacturing of gene therapies – our goal is to be responsive, flexible and action-oriented to ensure the business relationship enables the work we do.

To that end, we put you at the center of our work at every opportunity. Whether it is our dedicated team of project managers quality professionals, our regulatory support staff, or our commercial supply approach that reflects the potential curative nature of gene therapies, we hope you always feel the care we take in our work and the gratitude we have to be able to help bring these therapies to life.

At Viralgen, we want you to enable the fastest development of safe products in the gene therapy industry. To that end, our approach provides data early for planning and decision making, rapid access to capacity, and scales that meet your needs from ultra-rare to global demands.

We also know that AAV gene therapies must become more affordable to reach their full potential, and that the supply chain is complex and challenging to manage. To that end, we do not charge a royalty or milestones to use our platform, and take on much of the obligation for supply management and quality control as a part of our pricing model, which also includes robust regulatory support.

Regulatory support

Regulatory interactions in gene therapy are complex and can be challenging – as our understanding of AAV grows so too does the challenges of submission and approval of these therapeutics.

At Viralgen, we are committed to your success in those interactions with regulatory agencies. As a part of our inclusive pricing, we are happy to provide you with whatever level of support required for the Chemistry, Manufacturing and Control (CMC) areas of each submission – from providing template articles to authoring them, and participation in information exchanges with regulators either in writing or in person.

Life cycle management

Determining the right scale and right strategy for your clinical trials is challenging enough, but in a regulatory environment of dose escalations, multiple clinical endpoints and accelerated approval pathways, things get even more complicated.

On top of that, it’s an industry-wide aspiration to provide curative therapeutics, a situation that puts yet more complexity on how you as a client prepare for commercial launch, and commercial supply management.

Patient doses

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At Viralgen, we devote time and resource to engaging on these problems with you, and are happy to provide support to modeling scenarios together, taking dosing, trial design, and duration of therapeutic benefit into account as well as the fundamentals of the prevalence and incidence of each indication we support, and the competitive landscape around your therapeutic.

These models then inform flexible and creative commercial supply agreements, allowing demand, rather than our bottom line, to dictate supply arrangements with a focus on the cost of goods, rather than the batch price.

Of course, if you’re trying to change the world through using AAV in a pathway disease or oncology application we’re happy to support that too with modeling and supply that allows for long term growth.

Program management

Our project management team has deep experience in AAV and advanced therapies – from building and running large vector cores doctoral thesis on AAV biology, and working in commercialization of cell and gene therapies now on the market, their experience drives the success of our projects, and you benefit from academic, commercial and industrial experience.

Your program will be fully supported by a dedicated program manager.