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    Aspen Mori

    Research Analyst at Bank of America Merrill Lynch

    Aspen Mori's questions to Boundless Bio (BOLD) leadership

    Aspen Mori's questions to Boundless Bio (BOLD) leadership • Q1 2019

    Question

    Aspen Mori from Bank of America Merrill Lynch questioned the apparent disconnect between superior biopsy data in Cohort 2 and comparable functional scores for AT132 in XLMTM, and also asked about the competitive strategy for the DMD program.

    Answer

    Matt Patterson, Chairman and CEO, addressed the AT132 data by explaining that while biopsies show a dose-dependent effect, clinical measures have inherent variability, making it difficult to detect differences between doses when patients are achieving highly meaningful improvements and approaching normal function. For DMD, he stated the strategy is to be 'best-in-class' by producing a near-normal dystrophin protein, which they believe will prove superior to microdystrophin approaches over time. He emphasized that Audentes' manufacturing capabilities and a 'template approach' to development could accelerate timelines.

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    Aspen Mori's questions to Boundless Bio (BOLD) leadership • Q4 2018

    Question

    Aspen Mori, on behalf of Bank of America Merrill Lynch, questioned what the biopsy metrics looked like for XLMTM Cohort 2 and asked for the reason behind the screen failures in the Crigler-Najjar program.

    Answer

    President and CEO Matthew Patterson stated that detailed XLMTM biopsy data for Cohort 2 would be part of the next clinical update at ASGCT. Regarding Crigler-Najjar, he explained that screen failures in older teenage patients were due to unexpected baseline liver damage, which made them unsuitable for a Phase I/II trial despite their potential to benefit from the therapy.

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