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    Matthew [Analyst] • Bank of America

    Matthew [Analyst]'s questions to Regenxbio Inc (RGNX) leadership

    Matthew [Analyst]'s questions to Regenxbio Inc (RGNX) leadership • Q1 2025

    Question

    Speaking for Alec Stranahan, Matthew from Bank of America asked if there has been any change in the baseline characteristics of patients enrolling in the DMD trial and inquired about the target purity percentage for manufacturing.

    Answer

    President and CEO Curran Simpson reported that they are pleased with the breadth of patient ages enrolling in the DMD pivotal study, which should support a broad label. He stated that the manufacturing process achieves a purity level of 80% or greater with high consistency between batches, with more details to be presented at ASGCT.

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    Matthew [Analyst]'s questions to Regenxbio Inc (RGNX) leadership • Q3 2024

    Question

    Matthew [Analyst], on for Alec Stranahan at Bank of America, asked about the Duchenne program's durability in younger patients and redosing potential. He also inquired about the market impact of Eylea biosimilars on the RGX-314 wet AMD program.

    Answer

    Dr. Steve Pakola, CMO, addressed durability by pointing to evidence from their AAV8 serotype in other indications showing sustained effect despite cell division. On redosing, he said the current focus is on first-time treatment. Regarding biosimilars, he stated they are not seeing an impact on branded, longer-durability products, suggesting the value proposition of a one-time gene therapy like RGX-314 remains strong.

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