Freeline will present updated data on the use of analytical technologies for determining the proportion of AAV capsids containing full-length vector genomes, including the use of a panel of assays and combination of methodologies to fully characterise the AAV product. The ‘full-to-empty’ ratio discussed is an important quality attribute for AAVs with potential impact on safety and efficacy.
“We are pleased to report additional data on analytical technologies,” said Theresa Heggie, CEO. “This announcement comes in the final quarter of a pivotal year for Freeline, with a strengthened balance sheet following the successful launch of our IPO, continued progress in our clinical programmes in Haemophilia B and Fabry Disease, as well as advancements in our preclinical programmes in Gaucher Disease and Haemophilia A. It has always been our ambition to produce the best-in-class capsid, and these technologies will allow us to potentially increase safety and efficacy across our platform which will benefit all of our programmes.”
Title: Analytical Technologies for Determining the Proportion of AAV Capsids Containing Full-Length Vector Genomes
Presenter: Sonya Schermann, Director of Analytical Development at Freeline