Financial Data and Key Metrics Changes - The company has progressed significantly since its IPO five years ago, moving from no programs in the clinic to three programs in late-stage development [3][4] - The company plans to file three Biologics License Applications (BLAs) within a 12-month period, starting with the DMD Delzeta program for accelerated approval in the U.S. [4][24] Business Line Data and Key Metrics Changes - The Delzeta program has shown a 25% increase in dystrophin levels in patients, with a significant decrease in creatine kinase levels [11][12] - The company is focused on delivering first-in-class and best-in-class therapies across its product lines, with a strong emphasis on muscle delivery [4][10] Market Data and Key Metrics Changes - There are approximately 900 boys and young men in the U.S. with DMD exon 44, with about 50% of them treated at the same 40 centers of excellence [33] - The company anticipates that its therapies will be the first and best available options for DMD patients [34] Company Strategy and Development Direction - The company is preparing for commercialization with a foundational commercial infrastructure that can be leveraged across all three drug opportunities [9][10] - The company is exploring partnerships and M&A opportunities as it approaches the commercial phase with potential blockbuster drugs [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing their plans and emphasized the importance of focusing on upcoming approvals and launches [9][10] - The company is optimistic about the regulatory landscape and believes its data package will be compelling for the FDA [30][41] Other Important Information - The company has removed biopsy requirements for new participants in the Explore 44 trial, focusing on dystrophin and creatine kinase as key markers for the BLA filing [23][25] - The company is actively working on global trial enrollment for FSHD, with strong community support [46][47] Q&A Session Summary Question: Can you provide an overview of the company's platform and key programs? - The company has three programs in late-stage development and aims to improve lives through RNA therapy [3] Question: What is the commercial strategy and business development approach? - The company is building a commercial infrastructure and is open to exploring M&A opportunities [8][9] Question: Can you discuss the Delzeta program and its data? - The Delzeta program has shown a 25% increase in dystrophin levels and will present functional data ahead of schedule [11][12][16] Question: What is the market opportunity for DMD exon 44? - There are about 900 DMD exon 44 patients in the U.S., and the company plans to target this population effectively [33] Question: What are the key filing requirements for the BLA? - The primary requirements include demonstrating dystrophin levels and safety data, with a focus on creatine kinase as a critical marker [23][25] Question: Will there be a pre-BLA meeting? - Yes, a pre-BLA meeting is planned to ensure the data package meets FDA expectations [44] Question: What are the expectations for the Harbor study's primary endpoint? - The primary endpoint is video hand opening time, and the company is confident in meeting this goal [51]

Financial Data and Key Metrics Changes - The company has progressed significantly since its IPO five years ago, moving from no programs in the clinic to three late-stage development programs [3] - The company is on track to file three Biologics License Applications (BLAs) within a 12-month period, starting with the DMD Delzeta program for accelerated approval in the U.S. [4] Business Line Data and Key Metrics Changes - The Delzeta program for DMD has shown a 25% increase in dystrophin levels, rising from a baseline of 7% to 32%, and a decrease in creatine kinase (CK) to near-normal levels [11][12] - The company plans to present functional data for Delzeta ahead of schedule, indicating strong progress in its clinical trials [16] Market Data and Key Metrics Changes - There are approximately 900 boys and young men in the U.S. diagnosed with DMD exon 44, with about 50% of them treated at the same 40 centers of excellence [33] - The company anticipates being the first and best therapy available for DMD patients, leveraging its established patient services and field force [34] Company Strategy and Development Direction - The company is focused on building a foundational commercial infrastructure that can be leveraged across all three drug opportunities [10] - The strategy includes exploring partnerships and M&A opportunities as the company approaches the commercial phase with potential blockbuster drugs [7][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing the upcoming approvals and launches, emphasizing the importance of maintaining focus on their current programs [9] - The company is optimistic about the regulatory landscape, having aligned with the FDA on the requirements for accelerated approval based on dystrophin and CK data [24][26] Other Important Information - The company has initiated a biomarker study for the Delbrax program, which is expected to provide critical data for the accelerated approval pathway [40] - The company is actively working to ensure global trial enrollment for its FSHD program, indicating strong community interest and support [47] Q&A Session Summary Question: What drove the decision to report the data ahead of schedule? - The company aims to exceed expectations and found that the data was ready sooner than anticipated [16] Question: Can you give us a sense of patient number, follow-up time, and functional endpoints? - The upcoming data will involve 17 patients from the Explore 44 trial, with a focus on various functional measures [18][19] Question: Could you talk about the market opportunity for exon 44 and your go-to-market strategy? - The company sees a significant opportunity in the DMD market, with a well-established infrastructure to support patient access [33] Question: Will there be a pre-BLA meeting, and what will be the focus? - A pre-BLA meeting is planned to ensure the submission package meets FDA expectations [43] Question: What is the expectation for the primary endpoint in the Harbor study? - The company is confident in meeting the primary endpoint and is focused on a comprehensive data package for regulatory approval [52]