Ring Therapeutics presented new data at the ASCGT Conference in New Orleans showing that its AnelloVectors—produced using its AnelloBricks
in vitro assembly technology— have successful infectivity and expression of AnelloVectors. The data also demonstrates that the vectors can carry a wider range of payloads, expanding beyond the natural single-stranded DNA genome of anelloviruses.
To address the cumbersome and costly nature of traditional viral-based gene therapy manufacturing, Ring Therapeutics is developing AnelloBricks, which is a novel, cell-free, in vitro-assembled viral vector system based on commensal human anelloviruses. This system offers a modular, versatile, and scalable manufacturing platform solution for genetic medicines, according to Geoffrey Parsons, PhD, CSO of Ring Therapeutics. Enabled with only two components (a single recombinant capsid protein and a nucleic acid payload), AnelloBricks reduces manufacturing and cost, he added.
“Ring has demonstrated the ability to encapsidate nucleic acid payloads through in vitro assembly and achieved transduction in vitro and in vivo, showing significant progress toward developing a platform to manufacture a new class of functional viral vectors,” continued Parsons.
In an oral presentation (“AnelloBricks: Development of a Scalable, Low-Cost, In Vitro Assembled Anellovirus-Derived Platform for Gene Therapy Applications”), Parsons discussed the details around the development of AnelloBricks. Key points highlighted the potential of anellovirus-derived vectors as a novel gene therapy platform for a wide range of diseases, offering potential advantages in manufacturing scalability and cost compared to existing viral vector systems.
A poster presentation (“Infectivity and Expression in Mice of an Anelloviral Vector Produced by In Vitro Assembly”) represented the first demonstration of in vivo infectivity and expression of AnelloVectors assembled in vitro using Ring’s AnelloBricks platform. Transduction efficiency of AnelloBricks was improved by optimization of the production process (Production Process V1 vs Production Process V2) in HEK293TT cells resulting in a 10-fold increase in eGFP mRNA transcripts and a 10X increase in GFP protein.
Transduction efficiency of AnelloBricks in vivo in a rodent eye was improved when Production Process V2 was applied, resulting in a 10-fold increase in eGFP genome copies and mRNA transcripts compared to Production Process V1.
AnelloVectors produced from the AnelloBricks platform functionally delivered a cssDNA payload that resulted in confirmed mRNA and protein expression in both in vitro and in vivo models.
A second poster (“Purified Anelloviral Capsid Proteins Can Bind and Encapsidate Different Nucleic Acid Payloads into AnelloVectors for Gene Delivery”) focused on a study that showed the permissiveness of AnelloVectors to bind and encapsidate a diversity of nucleic acid payloads, including single and double stranded DNA and RNA with different structures and sizes.
Binding and encapsidation of different nucleic acid payloads with purified capsid proteins was determined by leveraging electrophoretic mobility shift assays and electron microscopy. Successful formation of AnelloVectors was achieved with both DNA and RNA, and several of these AnelloVectors were tested in in vitro transductions and found to be functionally active.
The post Ring Therapeutics Presents Promising New Data on its AnelloBricks Biomanufacturing Platform at ASCGT appeared first on GEN - Genetic Engineering and Biotechnology News.
To address the cumbersome and costly nature of traditional viral-based gene therapy manufacturing, Ring Therapeutics is developing AnelloBricks, which is a novel, cell-free, in vitro-assembled viral vector system based on commensal human anelloviruses. This system offers a modular, versatile, and scalable manufacturing platform solution for genetic medicines, according to Geoffrey Parsons, PhD, CSO of Ring Therapeutics. Enabled with only two components (a single recombinant capsid protein and a nucleic acid payload), AnelloBricks reduces manufacturing and cost, he added.
“Ring has demonstrated the ability to encapsidate nucleic acid payloads through in vitro assembly and achieved transduction in vitro and in vivo, showing significant progress toward developing a platform to manufacture a new class of functional viral vectors,” continued Parsons.
In an oral presentation (“AnelloBricks: Development of a Scalable, Low-Cost, In Vitro Assembled Anellovirus-Derived Platform for Gene Therapy Applications”), Parsons discussed the details around the development of AnelloBricks. Key points highlighted the potential of anellovirus-derived vectors as a novel gene therapy platform for a wide range of diseases, offering potential advantages in manufacturing scalability and cost compared to existing viral vector systems.
Poster presentations
A poster presentation (“Infectivity and Expression in Mice of an Anelloviral Vector Produced by In Vitro Assembly”) represented the first demonstration of in vivo infectivity and expression of AnelloVectors assembled in vitro using Ring’s AnelloBricks platform. Transduction efficiency of AnelloBricks was improved by optimization of the production process (Production Process V1 vs Production Process V2) in HEK293TT cells resulting in a 10-fold increase in eGFP mRNA transcripts and a 10X increase in GFP protein.
Transduction efficiency of AnelloBricks in vivo in a rodent eye was improved when Production Process V2 was applied, resulting in a 10-fold increase in eGFP genome copies and mRNA transcripts compared to Production Process V1.
AnelloVectors produced from the AnelloBricks platform functionally delivered a cssDNA payload that resulted in confirmed mRNA and protein expression in both in vitro and in vivo models.
A second poster (“Purified Anelloviral Capsid Proteins Can Bind and Encapsidate Different Nucleic Acid Payloads into AnelloVectors for Gene Delivery”) focused on a study that showed the permissiveness of AnelloVectors to bind and encapsidate a diversity of nucleic acid payloads, including single and double stranded DNA and RNA with different structures and sizes.
Binding and encapsidation of different nucleic acid payloads with purified capsid proteins was determined by leveraging electrophoretic mobility shift assays and electron microscopy. Successful formation of AnelloVectors was achieved with both DNA and RNA, and several of these AnelloVectors were tested in in vitro transductions and found to be functionally active.
The post Ring Therapeutics Presents Promising New Data on its AnelloBricks Biomanufacturing Platform at ASCGT appeared first on GEN - Genetic Engineering and Biotechnology News.