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CEO Vishwas Seshadri and CTO Brian Kevany addressed the CMC question, stating they aligned with the FDA in a Type A meeting and are confident in the resubmission package, with identity and Stargardt testing being the main new data points for FDA review. CCO Madhav Vasanthavada explained the commercial strategy, noting that market research shows high patient and physician interest in pz-cel due to its durable wound healing profile, and payers have not raised concerns about its use relative to other available therapies.

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CEO Vishwas Seshadri stated that no additional work was requested by the FDA, only input on the statistical approach for validation protocols. Chief Commercial Officer Madhav Vasanthavada explained the 3-month post-approval period is necessary for site-specific activities like P&T committee reviews and charge master updates, which require the final product label. Vasanthavada also reported that payer discussions have been positive, with payers recognizing pz-cel's distinct profile for large wounds and not raising major objections to its use alongside other treatments.

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Executive Alexander Sapir and Chief Scientific Officer Iain Fraser clarified that the healthy volunteer studies were always part of the routine development plan to assess formulations and drug interactions, and their timing was coincidental to the Oxbryta withdrawal. Regarding African sites, an executive (likely retiring CMO Pat Horn) stated the FDA has a long history of accepting ex-U.S. data that meets GCP standards, expressing confidence in the selected sites, many of which have prior regulatory audit experience.

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Executive Iain Fraser clarified that the powering for REACH was based specifically on the FSHD type 1 patient population from the ReDUX4 study. He explained that the over-enrollment of type 1 patients (from 210 to 242) is what increased the study's power from 93% to 96%. He noted that FSHD type 2 patients were stratified but not included in this primary power calculation.

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President and COO Peter Radovich stated the guidance increase is driven by continued strong Alagille syndrome growth and the robust PFIC launch in the U.S. Chief Medical Officer Joanne M. Quan confirmed the EXPAND study is on track with its previously guided 18-month enrollment timeline. CEO Christopher Peetz noted that while other indications for PDE4D may exist, the current focus is on achieving proof-of-concept in Fragile X.

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CMO David Lebwohl stated the trial will not include patients with major liver disease, as they are best treated by also reducing the mutant protein, which NTLA-3001 does not do. CEO John Leonard added that Intellia is developing a separate gene writing program to address both the lung and liver aspects of the disease.

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President and CEO Bradley Campbell clarified that the strategy is to first leverage their self-sustaining infrastructure to add de-risked products for top-line growth, with increased pipeline investment to follow as cash flow strengthens. Chief Development Officer Dr. Jeff Castelli confirmed the internal programs use refined and improved versions of the constructs from the Caritas work, with a primary focus on Fabry disease.

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President and CEO Bradley Campbell and Chief Development Officer Dr. Jeff Castelli confirmed this is a key focus. Their strategy is to shift physician expectations from disease stability to the possibility of improvement. Dr. Castelli added that Amicus reminds physicians that their clinical data showed patients improved after switching from prior ERTs, suggesting that 'stable' may not be the best possible outcome and that regaining function is a potential benefit of Pombiliti and Opfolda.

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CEO Emil Kakkis stated setrusumab is manufactured at a 3,000-liter scale at an approved and licensed contract manufacturer in Germany that they already use for Mepsevii. CCO Erik Harris and Kakkis confirmed that despite the Crysvita royalty transition, they have retained key commercial leadership and a field team of 16, providing a strong foundation to scale up for a successful launch.

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Chairman, CEO and President David Meeker said there are no current plans to share the RM-718 Phase I data but would consider it, and he was unsure about the extent of clinical trial site overlap. Chief Financial Officer Hunter Smith addressed the strategic question, stating it's too early to speculate on the trade-off but emphasized the company is dilution-sensitive while aiming to maximize long-term value.

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CEO John Evans noted that there is expected patient variability and that it is too early to draw firm conclusions. He pointed out a subtle variable for patients 3 and 4: their last blood transfusion was closer to the month-one time point, which can temporarily dilute the measurement of endogenously produced hemoglobin. He stated that longer follow-up is required to fully assess their trajectory.

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CEO John Evans noted that while there will be some patient variability, all patients showed robust early induction of HbF. He explained that for patients 3 and 4, the timing of their last blood transfusion relative to the one-month time point could affect early measurements, and suggested waiting for longer-term follow-up for a clearer comparison.

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CEO John Evans noted that some patient variability is expected and that for patients 3 and 4, their last blood transfusion was closer to the month 1 time point, which can temporarily dilute the measurement of endogenously produced hemoglobin. He advised that longer follow-up is needed to fully assess the trajectory and stabilization of hemoglobin levels.

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Christine Wilson, SVP and Head of U.S. Sales and Marketing, stated that success is built on balanced growth across both COEs and the community, noting that 70% of Q3 prescribers were new. President of R&D Suma Krishnan confirmed the year-end AATD update will include data from Cohorts 1 and 2. She added that if expression is sufficient in Cohort 2, they will stop there, but they have the option to proceed to Cohort 3 as no dose-limiting toxicities have been observed.

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Chief Scientific Officer Linda Burkly and CEO Gilmore O'Neill explained that indication selection is a pragmatic mix of targeting tissues with validated LNP delivery (like liver) and ensuring clinical translatability and commercial differentiation. Regarding competitors, O'Neill stated he is confident in reni-cel's differentiated profile, including its clinical data, manufacturing, and robust off-target editing profile from using AsCas12a.

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The company confirmed 18 patients have been dosed and stated there have been no dropouts, attributing any variation in dosing pace to normal scheduling fluctuations. They are on track for a mid-year data update. They have highlighted their potential differentiation to the FDA, but it is too early to comment on the agency's perception.

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The company believes their robust off-target analysis, which includes testing the drug product from many patients, is sufficient and shows no off-target editing. They see non-genotoxic conditioning as an important part of the field's evolution and are actively working in that area.

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CEO Craig Parker clarified that the SZN-043 study will enroll patients with chronic liver disease and fibrosis, who may not yet be considered cirrhotic. He stated there is no specific mechanistic mitigation strategy for transaminase elevations but noted that damaged tissue might be more sensitive, suggesting a dose-response approach. Parker added that while they won't disclose the exact number of SZN-1326 cohorts, the pharmacokinetic profile for SZN-043 was generally in line with animal projections, unlike SZN-1326.

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CEO Craig Parker clarified that the SZN-043 trial will enroll patients with chronic liver disease and fibrosis, who may not yet be considered frankly cirrhotic. He explained that there is no specific mechanistic mitigation strategy for transaminase elevations, but noted that preclinical data suggests damaged tissue may be more sensitive to the drug's regenerative effects, which could affect the dose-response. Parker confirmed that SZN-043's pharmacokinetics were in line with animal projections, unlike SZN-1326. He did not disclose the exact number of SZN-1326 dose cohorts but described it as a typical dose escalation strategy.

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CEO Gaurav Shah explained that the SAE reclassification was based on recent FDA guidance and is reflected in protocol changes. He affirmed that the strategy remains consistent with the May update, focusing on refined monitoring, SAE handling, safety guardrails, and eligibility criteria for earlier-stage patients.

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CMO Dr. Barbara White detailed that the top-line data will include safety, disposition, and primary and secondary endpoints for SSc, and the primary endpoint for CF. She explained that mRSS variability was minimized through rigorous investigator selection, training, and monitoring. Dr. White also affirmed a likely read-through from SSc to DM, as both use similar holistic composite scores with comparable components like physician/patient globals and key organ assessments.

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Executive Niels Riedemann explained that IFX-1 consumption in HS is about four times higher than in other undisclosed life-threatening diseases, likely due to a high C5a turnover rate, which aligns with KOL advice that high doses are needed in HS. He stated that proposing the IHS-4 score to regulators is supported by KOLs who believe it better captures all lesions, unlike HiSCR. To control future placebo response, he suggested using larger patient cohorts (80-100+ per group), avoiding 4:1 randomization, and potentially reducing trial touchpoints.

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