Financial Data and Key Metrics Changes - As of September 30, 2025, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for the ZEVASKYN commercial launch [15] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory and reclassification of certain costs to selling, general, and administrative expenses (SG&A) [16] - SG&A expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [16] - The net loss for Q3 2025 was $5.2 million, or -$0.10 per share, compared to a net loss of $30.3 million, or -$0.63 per share, in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ZEVASKYN commercial launch is progressing, with growing patient demand and the activation of a third qualified treatment center (QTC), Children's Hospital Colorado [5][12] - The number of identified eligible patients at QTCs has more than doubled to approximately 30, up from over 12 previously reported [11] - The company has received ZEVASKYN Product Order Forms (ZPOFs) for 12 patients, indicating strong interest and movement towards treatment [10] Market Data and Key Metrics Changes - ZEVASKYN has received coverage decisions from all major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs [13] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, which is expected to simplify claims and reimbursement processing [14] Company Strategy and Development Direction - The company is focused on scaling the ZEVASKYN commercial launch to meet patient demand and expanding its QTC network [5] - The management is actively discussing the onboarding of additional EB centers across the U.S. to further expand ZEVASKYN's geographic footprint [12] - The company aims to achieve profitability in the first half of 2026, despite the delay in the first patient treatment [30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2026 launch goals based on trends in patient demand and market access, despite a temporary delay in the first patient treatment [5][6] - The management highlighted the importance of maintaining high-quality standards in manufacturing personalized drug products [8] - The company is encouraged by the doubling of identified patients and the favorable payer landscape for ZEVASKYN [19] Other Important Information - The company has paused collecting additional patient biopsies to investigate and optimize a release assay that encountered performance issues [9] - The gene therapy program for X-linked retinoschisis, ABO-503, has been selected for the FDA Rare Disease Endpoint Advancement Pilot Program, which may accelerate development [18] Q&A Session Summary Question: Expected timeline for patients to receive treatment - Management indicated that several patients have already been scheduled for biopsy in November and early 2026, with treatment expected to follow if all paperwork is completed [24][26] Question: Impact on profitability timeline due to treatment delays - Management does not foresee a significant impact on the timeline for achieving profitability, maintaining guidance for the first half of 2026 [30] Question: Status of routine maintenance shutdown - The company confirmed a planned shutdown for routine maintenance starting mid-December and lasting about a month [34][35] Question: Biopsy collection during optimization pause - No biopsies were collected during the pause; the company prioritized resolving the assay issue before proceeding [36] Question: Lead time for ZEVASKYN Product Order Forms - Management expects the lead time to decrease as more patients progress through the treatment process and payer policies become established [39][42] Question: Attrition rate among patients with ZPOFs - Management anticipates a high conversion rate for patients with ZPOFs, as these are motivated patients who have expressed interest in treatment [47][49] Question: Prior authorization process for RDEB patients - The prior authorization process involves clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [52] Question: Revenue recognition timeline - Revenue is recognized when the product is administered to the patient, with cash flow considerations varying by site [58]

Financial Data and Key Metrics Changes - As of September 30, 2025, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for the ZEVASKYN commercial launch [15] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory and reclassification of certain costs to selling, general, and administrative expenses (SG&A) [16] - SG&A expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [16] - The net loss for Q3 2025 was $5.2 million, or negative $0.10 per share, compared to a net loss of $30.3 million, or negative $0.63 per share, in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ZEVASKYN commercial launch is progressing, with the first patient treatment delayed to Q4 2025 due to optimization of a product release assay, but demand is growing [5][6] - The number of identified eligible patients at qualified treatment centers (QTCs) has more than doubled to approximately 30, up from over 12 previously reported [11] - The activation of Children's Hospital Colorado as a QTC brings the total to three, alongside Lurie Children's Hospital of Chicago and Lucille Packard Children's Hospital Stanford [12] Market Data and Key Metrics Changes - ZEVASKYN has received positive coverage decisions from major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs effective October 1, 2025 [13] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, simplifying claims and reimbursement processing [14] Company Strategy and Development Direction - The company is focused on scaling the ZEVASKYN commercial launch to meet patient demand and expanding its QTC network [5][6] - The management team has been strengthened with the appointment of Dr. James A. Gao as Senior Vice President, Head of Clinical Development and Medical Affairs, to advance the pipeline [18] Management's Comments on Operating Environment and Future Outlook - Management remains confident in achieving 2026 launch goals despite the delay in the first patient treatment, citing trends in patient demand and market access [5][6] - The company expects to maintain a profitable business in the first half of 2026, with no significant impact from the treatment delay [29] Other Important Information - The company paused collecting additional patient biopsies to investigate a performance issue with a release assay, resuming in November 2023 [9] - The company plans to participate in the Stifel 2025 Healthcare Conference following the earnings call [16] Q&A Session Summary Question: Expected timeline for patients to receive treatment - Management indicated that several patients have already received prior authorizations and are scheduled for biopsies in November and early 2026 [24] Question: Impact on profitability timeline due to treatment delay - Management does not foresee a significant impact on the timeline to profitability, maintaining guidance for the first half of 2026 [29] Question: Routine maintenance shutdown of the plant - A shutdown for routine maintenance is scheduled for mid-December to early January, as mandated by the FDA [32] Question: Current lead time for ZEVASKYN Product Order Forms - The current lead time is about three months, but management expects this to decrease as more patients progress through the process [36] Question: Attrition rate among patients with ZEVASKYN Product Order Forms - Management expects a high conversion rate among patients who have received ZPOFs, as these are motivated patients [44] Question: Prior authorization process for RDEB patients - The prior authorization process involves clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [50] Question: Biopsies and sterility release criteria - Management confirmed that they have resumed biopsies and are not guiding on the number of biopsies completed yet [54] Question: Patients on background Vyjuvek or Filsuvez - Management does not have visibility into the exact number of patients currently on these treatments but expects many to be [58] Question: UMass not listed as a QTC - Different sites have various reasons for not being onboarded, including financial constraints and trade policy differences [62]

Financial Data and Key Metrics Changes - As of September 30, 2025, the company reported cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for operations over the next two years [13][14] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory [14] - Selling, general and administrative (SG&A) expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [15] - The net loss for Q3 2025 was $5.2 million, or negative $0.10 per share, compared to a net loss of $30.3 million, or negative $0.63 per share, in Q3 2024 [15] Business Line Data and Key Metrics Changes - The company is focused on the commercial launch of ZEVASKYN, the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), with growing patient demand and an expanding network of qualified treatment centers (QTCs) [4][10] - The number of identified eligible patients at QTCs has more than doubled to approximately 30, up from over 12 previously reported [10] - The activation of Children's Hospital Colorado as a QTC brings the total to three, alongside Lurie Children's Hospital of Chicago and Lucille Packard Children's Hospital Stanford [11] Market Data and Key Metrics Changes - ZEVASKYN has received coverage decisions from all major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs effective October 1, 2025 [12] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, simplifying claims and reimbursement processing [12] Company Strategy and Development Direction - The company aims to achieve its 2026 launch goals for ZEVASKYN despite a temporary delay in the first patient treatment, focusing on expanding treatment center networks and ensuring market access [4][5] - The company is also advancing its pipeline, with a gene therapy program for X-linked retinoschisis selected for the FDA Rare Disease Endpoint Advancement pilot program [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to meet 2026 launch goals based on trends in patient demand and treatment center expansion, despite the delay in the first patient treatment [4][5] - The management team highlighted the importance of maintaining high-quality standards in manufacturing and the proactive measures taken to optimize release assays [6][7] Other Important Information - The company has strengthened its management team with the appointment of Dr. James A. Gao as Senior Vice President, Head of Clinical Development and Medical Affairs, bringing over 20 years of industry experience [17] Q&A Session Summary Question: Expected timeline for patients to receive treatment after ZEVASKYN Product Order Forms - Management indicated that many patients have already received prior authorizations and are scheduled for biopsies in November and early 2026, with treatment expected to follow if all paperwork is completed [20][22] Question: Impact of treatment delays on profitability timeline - Management stated that the timeline for achieving profitability remains unchanged, with expectations for profitability in the first half of 2026 [26][28] Question: Routine maintenance shutdown of the plant - Management confirmed a mandated FDA-required shutdown for general maintenance from mid-December to early January [29][30] Question: Status of biopsies collected before optimization pause - Management clarified that no biopsies were collected during the pause to ensure patient safety and product quality [31] Question: Lead time for ZEVASKYN Product Order Forms - Management expects the lead time to decrease as more patients progress through the treatment process, currently estimated at about three months [34] Question: Attrition rate among patients with ZEVASKYN Product Order Forms - Management expressed confidence in a high conversion rate for patients with ZPOFs, as these are motivated patients eager to proceed with treatment [40][41] Question: Prior authorization process for RDEB patients - Management described the prior authorization process as involving clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [44][46]