Klaudia Kuranda

Klaudia Kuranda

Company: Spark Therapeutics

Job title: Head of Immunology




Modulation of the Innate Response to AAV Gene Therapy Vectors 11:00 am

Since potentially immune-related toxicities have been observed in AAV gene therapy trials, there is a need for identification of efficacious immunomodulatory strategies We describe innate immune responses to AAV and identify the key role of preexisting antibodies, complement and IL-6 signalling pathways Short-term inhibition of the early innate responses at the time of vector administration…Read more

day: Day Two

Panel Discussion: Uncovering Unknowns About the Ratio of Percentage Full Vs Percentage Empty Capsids On Safety & Efficacy of AAV Gene Therapy 3:15 pm

What is the impact of percentage full vs empty ratio on efficacy and safety? What role does this play on immunogenicity of the gene therapy? What are the acceptable limits of empty capsids in a drug product and which assays are use to measure it? Chaired by: Sam Wandsworth, Chief Scientific Officer, UltragenyxRead more

day: Day One

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