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    Tiffany Marchell

    Research Analyst at William Blair

    Tiffany Marchell's questions to SELB leadership

    Tiffany Marchell's questions to SELB leadership • Q4 2022

    Question

    Tiffany Marchell from William Blair asked about the prophylactic use of the IgG Protease Xork with Astellas for pre-existing antibodies, including potential efficacy cutoffs, and inquired if more preclinical data on Xork would be shared this year.

    Answer

    President and CEO Carsten Brunn explained that the general approach for Xork is to treat patients with pre-existing antibodies from natural AAV infections, which could expand the eligible patient pool by 30-70%. He noted this was a key factor in the Astellas deal. He also stated that the company has not guided on sharing additional preclinical data for Xork at this time.

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    Tiffany Marchell's questions to SELB leadership • Q4 2022

    Question

    Tiffany Marchell from William Blair asked about the planned prophylactic use of the IgG Protease Xork with Astellas, whether there would be a cutoff for pre-existing antibody levels, how much Xork could expand the treatable patient population, and if more preclinical data would be shared this year.

    Answer

    President and CEO Carsten Brunn explained that Xork is intended to address pre-existing immunity from natural AAV infections, which affects 30-70% of the population, potentially doubling the number of eligible patients for gene therapy. He could not comment on Astellas' specific development plans and stated that the company has not guided on sharing additional preclinical data for Xork at this time.

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    Tiffany Marchell's questions to SELB leadership • Q3 2022

    Question

    Tiffany Marchell of William Blair inquired about plans to share data from the IL-2 and IgA protease selection processes and asked for an update on activities required to initiate the MMA gene therapy study.

    Answer

    President and CEO Carsten Brunn stated that data on the selected candidates and the selection process will be shared at scientific conferences in the coming year. Chief Medical Officer Peter Traber confirmed that all necessary approvals for the MMA study are in place, and initiation with patient screening is imminent.

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