AAV Curator™ Platform

AAV Curator™ Platform logo

Andelyn's Suspension and Adherent Modular Platform Process

Optimization-By-Design™

Industrializing Gene Therapy Processes with a Reductionist, Holistic and Systematic Optimization Methodology Approach

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Configurable
Modular platform with assessment and strategy adapted to each program drives faster optimization.
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Optimization Toolkit
QbD guidelines and DoEs with lean, step-wise focus on process understanding maximizes program efficiency.
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Data Driven
Historical data leveraged to achieve high yield and high-quality products with predictable performance.

Consistency in productivity and quality are evaluated at each stage of development and measured with enabling technologies.

AAV Curator™ Platform logo

Built on data and configurability and ensures the end process is curated to your program's specific needs to optimize yields.

Development of any platform process should account for optimization variables across upstream, downstream and fill and finish unit operations, materials and quality control testing panels and quality assurance release parameters. Our modular approach uses configurable materials and methods optimized for your process requirements and reinforced with data and analytics.

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  • Adherent process is a low serum system utilizing a fortified calf serum (no FBS) with progressive reduction through the production process.
  • Suspension process utilizes chemically defined serum-free media for ultimate flexibility of scale across multiple bioreactor platforms.
  • Standard Bill of Materials including off-the-shelf (OTS) products that drive reduced lead times and security of supply, full QC testing.

AAV Curator™ Cell Line

Clonal HEK293 cell line for high yield AAV production

AAV Curator Cell Line chart

Andelyn's Curator™ Cell Line is a clonal HEK293 cell line that improves rAAV titers across multiple serotypes.

  • DoE was used to systematically improve rAAV titers for multiple serotypes
  • Productivity assessed at harvest by collecting and testing supernatant
  • Titers of vg/ml as determined by ddPCR
Chart showing Optimal growth at high Viable Cell Density (VCD) to maximize vector production.

Optimal growth at high Viable Cell Density (VCD) to maximize vector production.

  • The red region of the response contour plot indicates the design space which results in the highest overall yields of rAAV under the range of conditions investigated.
  • Reduced DNA/cell in conjunction with optimal cell density to increase titer maximizes productivity.

Full Program Partnering

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full program partnering chart

Benefits of Our Platform Approach

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Savings

Reduced Spending on Optimization

High Productivity and % Full Capsids driving production costs down

No MCB Generation Costs

No Tech Transfer Costs

No PreClinical/clinical Licensing Fees

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Expedited Timelines

Reduced time spent on opTimization

Off the Shelf (OTS) plasmids readily avaIlable

No MCB generatIon time

Supply Chain Already Established

NO TECH TRANSFER/DOCUMENTATION TIME

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Smooth CMC

DMFs filed for Andelyn’s platform

Same equipment in non-GMP & GMP

Same quality systems

Integrated teams in Preclinical & Clinical

Hear from Our Partners

Success Stories
Learn More About Us
“Working with Andelyn was a great choice given their history with the product, their extensive multi-product experience with the manufacturing technology used for the product, and our desire to quickly bring UX111 to MPS IIIA patients.”
Dennis Huang
Chief Technical Operations Officer and EVP, Gene Therapy Research and Development
Ultragenyx
“Purespring is excited to build on our longstanding working relationship with Andelyn to accelerate the preclinical and clinical manufacture of our innovative AAV-based gene therapies.”
Julian Hanak
CEO
Purespring Therapeutics

Does your program use another vector rather than AAV?

Check out our Tech Transfer capabilities.

Learn More
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