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AAVantage® Novel AAV Capsid Products

Porton Advanced has accumulated unique experience in designing highly complex AAV capsid libraries, using strong structural biology and bioinformatics expertise. Unlike traditional library design methods, our multi-step construction approach allows us to alter the capsid surface with high precision on a large scale, while maximizing variant compatibility with capsid assembly. The resulting AAV capsid libraries have large numbers of functional variants (over 1E8) with significantly altered viral tropism and immunity evasion potential.

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Evolution and Screening Methods

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Ways to Collaborate

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Ways to Collaborate

Using AAVantage®-Novel AAV Capsid Evolution and Screening Platform , we have designed and validated several capsid libraries, and developed novel AAV capsid variants for retina and liver-directed human gene therapy. 

Services

Porton Advanced designs and performs all necessary R&D steps ending with the delivery of a fully characterized novel capsid with improved properties that fit the customer’s needs.

  • Criteria can include tissue specificity, immune evasion, transduction efficiency, production yield, and even packaging capacity.
  • In addition to our existing capsid libraries, new libraries can be designed based on any particular serotype or variant.
  • Library screening can be performed in vitroor in vivo, or using a combination of both.

Customers with their own capsid engineering projects can outsource any portion of their workflow to Porton Advanced, such as:

  • Capsid library construction, production, and quality control
  • In vitro selection using 3D cultures.
  • In vivoselection using non-human primates (NHP) or humanized / xenografted mice.
  • NGS and data analysis, including Illumina or PacBio sequencing, with identification of most promising candidates or recommendation for further selection or diversification.
  • Variant characterization, including in vitro(transduction efficiency, immune evasion, production yield), and in vivo (biodistribution).

AAVantage® Complete Solution

Porton Advanced designs and performs all necessary R&D steps ending with the delivery of a fully characterized novel capsid with improved properties that fit the customer’s needs.

  • Criteria can include tissue specificity, immune evasion, transduction efficiency, production yield, and even packaging capacity.
  • In addition to our existing capsid libraries, new libraries can be designed based on any particular serotype or variant.
  • Library screening can be performed in vitroor in vivo, or using a combination of both.

AAVantage® Custom Services

Customers with their own capsid engineering projects can outsource any portion of their workflow to Porton Advanced, such as:
  • Capsid library construction, production, and quality control
  • In vitro selection using 3D cultures.
  • In vivoselection using non-human primates (NHP) or humanized / xenografted mice.
  • NGS and data analysis, including Illumina or PacBio sequencing, with identification of most promising candidates or recommendation for further selection or diversification.
  • Variant characterization, including in vitro(transduction efficiency, immune evasion, production yield), and in vivo (biodistribution).

Products

Retina-targeting AAV capsid variants

  • High tissue specificity for RPE
  • Intravitreal delivery
  • Selection in NHP
  • Transduction efficiency 5x better than AAV2-7m8

Liver-targeting AAV capsid variants with exceptional immune evasion properties

  • High specificity for human hepatocytes
  • Better transduction efficiency than the current best variant AAV3B-V04
  • Exceptional evasion of preexisting neutralizing antibodies
  • 3 libraries based on human serotypes
    • High transduction efficiency and production yield
  • 2 library based on a non-primate AAV
    • Evasion of human preexisting neutralizing antibodies
  • 1 library based on a non-AAV parvovirus
    • Large capacity of genome size (up to 6.1 kb)

AAVantage®- ready to use AAV capsid variants and capsid libraries

Novel AAV capsid for retinal pigmented epithelium (RPE) targeting via intravitreal injection

Retina-targeting AAV capsid variants

  • High tissue specificity for RPE
  • Intravitreal delivery
  • Selection in NHP
  • Transduction efficiency 5x better than AAV2-7m8

Novel AAV capsid for retinal pigmented epithelium (RPE) targeting via intravitreal injection

Liver-targeting AAV capsid variants with exceptional immune evasion properties

  • High specificity for human hepatocytes
  • Better transduction efficiency than the current best variant AAV3B-V04
  • Exceptional evasion of preexisting neutralizing antibodies

Capsid libraries

  • 3 libraries based on human serotypes
    • High transduction efficiency and production yield
  • 2library based on a non-primate AAV
    • Evasion of human preexisting neutralizing antibodies
  • 1library based on a non-AAV parvovirus
    • Large capacity of genome size (up to 6.1 kb)
Four different colored coronaviruses on a white background.

Flexible Business Models

Porton Advanced is open to explore R&D partnerships on projects of mutual interest