Abeona Therapeutics (NasdaqCM:ABEO) FY Conference September 09, 2025 02:00 PM ET Company ParticipantsMadhav Vasanthavada - CCODaniel Smith - Equity Research Associate in BiotechnologyDaniel SmithGood afternoon, everyone, and thank you for joining Day 2 of the H.C. Wainwright 27th Annual Global Investment Conference 2025. My name is Daniel Smith, and I'm an H.C. Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our presenters for the session. I'd like to welcome Madhav V ...

Summary of Abeona Therapeutics (ABEO) 2025 Conference Call Company Overview - Abeona Therapeutics is a commercial-stage biotech company based in Cleveland, specializing in cell and gene therapy, particularly for patients with recessive dystrophic epidermolysis bullosa (RDEB) [4][5] - The company has launched its lead product, ZivaSkin, which is an autologous cell-based gene therapy aimed at treating large, chronic wounds associated with COL7 deficiency [4][5] Key Highlights - **Product Launch and Demand**: - ZivaSkin has shown strong initial demand with approximately 50 patients identified for treatment, even before a dedicated sales force was established [5][6] - The company has two qualified treatment centers (QTCs) operational, with plans to expand further [22][24] - **Patient Profile**: - The initial patient cohort includes severe cases with large, chronic wounds, many of whom are treatment naive or have previously tried other therapies [7][9][36] - An estimated 750 patients with severe and moderate wounds are eligible for ZivaSkin across 23 centers of excellence [9][10] - **Clinical Evidence**: - ZivaSkin's clinical trials have demonstrated unique efficacy in treating chronic wounds that have remained open for extended periods, with some patients experiencing wounds that have not healed for up to 21 years [12][13] - The therapy aims to provide durable wound healing and pain reduction after a single application [4][12] - **Manufacturing and Capacity**: - Initial manufacturing capacity is set at two patients per QTC per month, with plans to ramp up to 10 patients per month by mid-2026 [22][50] - The company is preparing for potential scalability in manufacturing to meet increasing demand [50][51] Market Potential and Financial Outlook - **Patient Demand and Market Potential**: - The company anticipates treating 10 to 14 patients in 2025, with a strong pipeline of referrals already in place [26][29] - The process from patient identification to treatment is expected to take about four months initially, with improvements anticipated as the process becomes more streamlined [30][32] - **Reimbursement Landscape**: - Positive trends in payer coverage have been observed, with all prior authorizations submitted so far being approved, some within 48 hours [41][42] - Approximately 60% of patients are commercially insured, with favorable coverage policies emerging from major payers like United Health [43][45] Additional Insights - **Patient Experience and Logistics**: - The treatment process involves multiple administrative steps, including insurance clearance and biopsy collection, which are expected to improve over time [17][18] - Patients are willing to travel significant distances to access treatment, indicating a strong commitment to receiving ZivaSkin [24][25] - **Future Developments**: - Abeona is also developing proprietary capsids for retinal inherited diseases, with clinical trials expected to start in late 2026 [55] - The company emphasizes the importance of execution in the current phase, with a strong balance sheet supporting its operations [54] Conclusion - Abeona Therapeutics is positioned for growth with its innovative ZivaSkin therapy, strong initial demand, and positive reimbursement trends. The company is focused on expanding its treatment capacity and enhancing patient access while preparing for future product developments in its pipeline.

Group 1 - The article discusses the offerings of Biotech Analysis Central, which provides in-depth analysis of pharmaceutical companies and a model portfolio of small and mid-cap stocks [1][2] - The service includes over 600 biotech investing articles and live chat features to assist healthcare investors in making informed decisions [2] - A promotional offer is available for new subscribers, including a two-week free trial and a discounted annual subscription price [1] Group 2 - The author emphasizes that there are no current stock or derivative positions in any mentioned companies, ensuring an unbiased perspective [3] - The article clarifies that past performance does not guarantee future results, and no specific investment recommendations are provided [4]

Financial Data and Key Metrics Changes - As of June 30, 2025, the company reported unaudited cash, cash equivalents, short-term investments, and restricted cash of $225.9 million, a significant increase from $98.1 million as of December 31, 2024, providing over two years of operating capital without further capital infusion [15][16] - Net income for the second quarter of 2025 was $108.8 million, or $2.07 per basic share, compared to a net income of $7.4 million, or $0.19 per basic share, in the same quarter of 2024 [18] Business Line Data and Key Metrics Changes - The company has seen strong interest in ZevaSkin, with over 50 identified patients across two qualified treatment centers (QTCs) and expectations to treat 10 to 14 patients in 2025 [14][5] - Research and development expenses decreased to $5.9 million for the quarter ended June 30, 2025, from $9.2 million in the same quarter of 2024, primarily due to costs capitalized into inventory [17] Market Data and Key Metrics Changes - The company has achieved positive coverage with multiple national and regional payers, with 100% of prior authorization requests approved to date, indicating strong clinical acceptance among payers [11][12] - Approximately 60% of RDEB lives are covered by commercial plans, 30% by Medicaid, and the rest by Medicare, highlighting the market potential for ZevaSkin [10] Company Strategy and Development Direction - The company is focused on expanding its geographic footprint by activating additional QTCs to ease travel burdens for patients and increase demand for ZevaSkin [8][14] - The company plans to transition into a revenue-generating commercial entity and will provide high-level forward cost guidance alongside updates on commercialization progress [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch progress of ZevaSkin, citing positive feedback from the RDEB community and the potential to improve patient lives [4][14] - The company anticipates that the first ZevaSkin patient treatment will occur in 2025, leading to projected company-wide profitability in early 2026 [16] Other Important Information - The company has partnered with Deborah of America for a nationally broadcast webinar to share patient experiences, which has positively influenced community engagement [13] - The company is exploring opportunities for EU and Japan market entries, considering the feasibility of supplying from its Cleveland facility [77] Q&A Session Summary Question: How are identified patients defined? - Identified patients are those with severe wounds that have never healed, prioritized by physicians for treatment with ZevaSkin [24][25] Question: What is the treatment timeline for patients outside the two QTCs? - Patients have started the referral process and are going through initial consultations, with plans to activate additional treatment centers to facilitate their treatment [26][27] Question: How many cases does a center need to treat before adopting ZevaSkin as routine therapy? - There is no specific number of pre-treatments required; centers are confident in treating patients based on their experience with RDEB patients [33][36] Question: What are the payment mechanics for ZevaSkin? - Revenue is recognized after a patient has been treated with ZevaSkin, with hospitals able to secure payer agreements before placing orders [49][51] Question: What is the status of prior authorizations? - The company has seen a 100% approval rate for prior authorizations, with no pushback from payers regarding prior treatments before approving ZevaSkin [58][55] Question: What are the production capacity plans? - The company is on track to ramp up production capacity to treat 10 patients per month by mid-2026, with no anticipated FDA inspections affecting this timeline [64][68] Question: What are the expectations for the EU market? - The company is exploring the EU market but acknowledges that establishing a manufacturing footprint there would take significant time [77]

[PART I - FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed consolidated financial statements for June 30, 2025, reflect significant financial improvement, driven by a $152.4 million PRV gain and resulting in $96.8 million net income [Condensed Consolidated Balance Sheets](index=5&type=section&id=Unaudited%20Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets significantly increased to $246.2 million, primarily due to a rise in cash and cash equivalents, leading to positive stockholders' equity Balance Sheet Highlights ($ in thousands) | Item | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $163,535 | $23,357 | | Total current assets | $232,262 | $100,853 | | Total assets | $246,233 | $108,931 | | **Liabilities & Equity** | | | | Total liabilities | $82,657 | $64,900 | | Total stockholders' equity | $163,576 | $44,031 | | Total liabilities and stockholders' equity | $246,233 | $108,931 | [Condensed Consolidated Statements of Operations and Comprehensive Income (Loss)](index=6&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Income%20%28Loss%29) For the six months ended June 30, 2025, the company achieved a net income of $96.8 million, a significant turnaround from a $24.2 million loss in 2024, primarily due to a $152.4 million PRV gain Income Statement Highlights ($ in thousands) | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | License and other revenues | $400 | $0 | | Research and development | $15,884 | $16,425 | | Selling, general and administrative | $26,935 | $15,769 | | Loss from operations | ($42,519) | ($32,194) | | Gain from sale of priority review voucher, net | $152,366 | $0 | | Net income (loss) | $96,804 | ($24,172) | | Basic income (loss) per common share | $1.89 | ($0.72) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2025, net cash increased by $140.2 million to $163.9 million, driven by $160.1 million from investing activities, primarily the PRV sale Cash Flow Summary ($ in thousands) | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($37,186) | ($27,222) | | Net cash provided by (used in) investing activities | $160,101 | ($51,949) | | Net cash provided by financing activities | $17,263 | $99,124 | | Net increase in cash, cash equivalents and restricted cash | $140,178 | $19,953 | | Cash, cash equivalents and restricted cash at end of period | $163,873 | $34,764 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's transition to a commercial-stage entity, highlighting the $152.4 million PRV sale, initial license revenue, inventory capitalization, and sufficient capital for the next 12 months - On April 28, 2025, the FDA approved ZEVASKYN™ for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB), marking the company's transition to a commercial-stage biopharmaceutical company[25](index=25&type=chunk) - Following FDA approval, the company began capitalizing inventory costs related to ZEVASKYN™ As of June 30, 2025, inventory consisted of **$2.7 million** in raw materials[32](index=32&type=chunk)[13](index=13&type=chunk) - In June 2025, a third party exercised an option to license certain of the company's AAV capsids, resulting in **$0.4 million** of license revenue The agreement includes potential future milestone and royalty payments[97](index=97&type=chunk)[99](index=99&type=chunk) - The company sold a Priority Review Voucher (PRV) in June 2025, receiving gross proceeds of **$155.0 million** and recognizing a net gain of **$152.4 million** after transaction costs[118](index=118&type=chunk) - The company recorded an income tax expense of **$15.5 million** for the first half of 2025, driven by the pre-tax income from the PRV sale The company is assessing the impact of new tax legislation which may reduce its 2025 tax liability[114](index=114&type=chunk)[117](index=117&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=31&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's transition to a commercial-stage entity, highlighting the $155.0 million PRV sale, ZEVASKYN™ commercial launch preparations, and sufficient liquidity for the next 12 months - Abeona is now a commercial-stage company after the FDA approved ZEVASKYN™ on April 28, 2025, for treating wounds in RDEB patients[121](index=121&type=chunk) - The company sold its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of **$155.0 million**, with the transaction closing on June 27, 2025[122](index=122&type=chunk) - Commercial launch of ZEVASKYN™ is underway, with the first Qualified Treatment Center (QTC) activated in May 2025 and treatments expected to start in Q3 2025[123](index=123&type=chunk) - As of June 30, 2025, cash resources were **$225.9 million**, which management believes is sufficient to fund operations for at least the next 12 months[167](index=167&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) For Q2 2025, net income reached $108.8 million, primarily due to the $152.4 million PRV gain, while R&D decreased 36% and SG&A increased 98% due to commercialization efforts Financial Performance ($ in thousands) | Item | Q2 2025 | Q2 2024 | Change (%) | | :--- | :--- | :--- | :--- | | License and other revenues | $400 | $0 | 100% | | Research and development | $5,943 | $9,218 | (36)% | | Selling, general and administrative | $17,149 | $8,646 | 98% | | Gain from sale of PRV, net | $152,366 | $0 | 100% | | Net income | $108,833 | $7,406 | 1,370% | - The decrease in Q2 R&D spending was primarily due to **$1.4 million** of costs capitalized into inventory and **$4.9 million** of production costs reclassified as SG&A following ZEVASKYN™'s FDA approval[132](index=132&type=chunk) - The increase in Q2 SG&A expenses was driven by approximately **$4.9 million** in reclassified production costs and a **$3.7 million** increase in salaries, stock-based compensation, and other professional fees related to commercial launch preparations[134](index=134&type=chunk) [Liquidity and Capital Resources](index=36&type=section&id=Liquidity%20and%20Capital%20Resources) The company's liquidity significantly improved to $225.9 million as of June 30, 2025, primarily from the $155.0 million PRV sale, with current capital sufficient for the next 12 months Cash Flow Summary ($ in thousands) | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($37,186) | ($27,222) | | Net cash provided by (used in) investing activities | $160,101 | ($51,949) | | Net cash provided by financing activities | $17,263 | $99,124 | - The company's principal source of liquidity is its **$225.9 million** in cash resources as of June 30, 2025, which includes the proceeds from the PRV sale[167](index=167&type=chunk) - During the first six months of 2025, the company raised **$17.3 million** in net proceeds from selling **3,510,889 shares** under its ATM Agreement As of June 30, 2025, up to **$51.5 million** remains available for sale under the agreement[168](index=168&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section is not applicable for the current reporting period - The company has indicated that quantitative and qualitative disclosures about market risk are not applicable[178](index=178&type=chunk) [Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal control over financial reporting during the quarter - The Chief Executive Officer and Chief Financial Officer concluded that the company's Disclosure Controls and Procedures were effective as of June 30, 2025[180](index=180&type=chunk) - No changes occurred during the quarter ended June 30, 2025, that have materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[181](index=181&type=chunk) [PART II - OTHER INFORMATION](index=40&type=section&id=PART%20II%20-%20OTHER%20INFORMATION) [Legal Proceedings](index=40&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the current period - There are no legal proceedings to report[184](index=184&type=chunk) [Risk Factors](index=40&type=section&id=Item%201A.%20Risk%20Factors) The company highlights new risks related to commercializing its first product, ZEVASKYN™, including revenue generation challenges, NOL limitations, and potential government agency disruptions - As a newly commercial-stage company with its first approved product, ZEVASKYN™, it is difficult to assess future viability, and the company may not be able to sustain profitability[186](index=186&type=chunk) - The company's ability to generate significant revenue depends on the successful commercialization of ZEVASKYN™, which involves overcoming challenges in market acceptance, reimbursement, and patient identification[187](index=187&type=chunk)[188](index=188&type=chunk) - The company's ability to use its **$416.1 million** in U.S. federal net operating loss (NOL) carryforwards may be materially limited due to multiple historical ownership changes under Section 382, with a full analysis expected by year-end 2025[191](index=191&type=chunk)[192](index=192&type=chunk) - Disruptions at the FDA and other government agencies, such as those caused by funding shortages, hiring freezes, or reorganization, could delay or prevent product approvals and commercialization, negatively impacting the business[194](index=194&type=chunk) [Unregistered Sale of Equity Securities and Use of Proceeds](index=42&type=section&id=Item%202.%20Unregistered%20Sale%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) During Q2 2025, the company reported the surrender of **885** common shares at **$6.24** per share to satisfy tax withholding obligations from restricted stock vesting Shares Surrendered for Tax Withholding | Period | Shares Purchased | Average Price Paid per Share ($) | | :--- | :--- | :--- | | June 1, 2025 - June 30, 2025 | 885 | $6.24 | | **Total Q2 2025** | **885** | **$6.24** | - The shares were surrendered to the company to cover tax withholding obligations from the vesting of restricted stock awards[199](index=199&type=chunk) [Other Information](index=43&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during Q2 2025 - No directors or executive officers adopted, modified, or terminated any Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during the second quarter of 2025[201](index=201&type=chunk) [Exhibits](index=43&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including the PRV Asset Purchase Agreement, officer certifications, and interactive data files - Key exhibits filed include the Priority Review Voucher Asset Purchase Agreement dated May 9, 2025, and certifications from the Principal Executive Officer and Principal Financial Officer[207](index=207&type=chunk)

Financial Transactions - Abeona Therapeutics completed the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $155 million on June 27, 2025[6]. - As of June 30, 2025, the company's unaudited cash, cash equivalents, restricted cash, and short-term investments totaled approximately $225 million, including net proceeds from the asset sale[8][10]. - The asset sale was executed under a purchase agreement dated May 9, 2025, specifically for the PRV[6]. - The press release regarding the asset sale was issued on July 2, 2025, highlighting the completion of the transaction[8]. Regulatory and Compliance - The Priority Review Voucher was awarded to the company on April 28, 2025, following the approval of its biologics license application for ZEVASKYN™[5]. - The financial information provided is preliminary and subject to completion of the company's financial closing procedures[11]. - The independent registered public accounting firm has not reviewed the preliminary estimates and does not provide any assurance regarding them[11]. - The company plans to file the full text of the PRV Asset Purchase Agreement with its Quarterly Report for the three months ended June 30, 2025[7]. Strategic Initiatives - The asset sale is part of the company's strategy to enhance its financial position and support future growth initiatives[6]. - The company is classified as an emerging growth company under the Securities Act of 1933[4].

Core Insights - Abeona Therapeutics has received FDA approval for ZEVASKYN™, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][4] - The U.S. launch is on track, with the first patient treatment expected in the third quarter of 2025, supported by strong patient interest and positive insurance coverage [1][2] - As of June 30, 2025, the company reported $226 million in cash and equivalents, expected to fund operations for over two years before ZEVASKYN revenue begins [1][5] Recent Developments - ZEVASKYN was approved by the FDA in April 2025 and is now available at Qualified Treatment Centers (QTCs) [4] - Strong demand for ZEVASKYN has been noted, with over a dozen patients identified for treatment and 100% approval of prior authorization requests submitted [4] - The company has established positive coverage with major insurers, including United Healthcare, covering approximately 16% of the U.S. insured population [4] Financial Results - For the second quarter of 2025, Abeona reported a net income of $108.8 million, significantly up from $7.4 million in the same period of 2024 [8] - Research and development expenses decreased to $5.9 million from $9.2 million year-over-year, primarily due to reclassification of costs [7] - Selling, general, and administrative expenses increased to $17.1 million, reflecting the costs associated with the commercial launch of ZEVASKYN [7] Operational Progress - The company is on track to ramp up ZEVASKYN supply capacity to treat up to 10 patients per month by mid-2026 [4] - Abeona has entered into a licensing agreement for the AAV204 capsid for potential gene therapies in ophthalmology [4] - The company secured $155 million from the sale of its Rare Pediatric Disease priority review voucher, awarded upon FDA approval of ZEVASKYN [4]

Core Insights - AscellaHealth has partnered with Abeona Therapeutics to support the commercialization of ZEVASKYN™, an FDA-approved cell-based gene therapy for treating recessive dystrophic epidermolysis bullosa (RDEB) [1][2][10] - The partnership focuses on creating patient-centric solutions that address clinical, operational, and reimbursement needs, enhancing patient access and clinical outcomes [1][2][9] Company Overview - AscellaHealth provides customizable solutions for the specialty pharmaceutical industry, emphasizing patient support and efficient healthcare delivery [1][9] - Abeona Therapeutics is a biopharmaceutical company developing innovative cell and gene therapies, with ZEVASKYN™ being its flagship product for RDEB [10] Product Details - ZEVASKYN™ is the first autologous cell sheet-based gene therapy for RDEB, utilizing the COL7A1 gene to produce functional type VII collagen in patients' skin cells [3][5][6] - The therapy has shown clinically meaningful results in wound healing and pain reduction with a single application [5][6] Partnership Dynamics - The collaboration between AscellaHealth and Abeona Therapeutics is characterized by shared goals, open communication, and a commitment to patient-centric care [2][9] - AscellaHealth's HUB model has proven effective in enhancing compliance, retention, and satisfaction rates for specialty pharmaceuticals and gene therapies [2]

Core Insights - Abeona Therapeutics has activated a Qualified Treatment Center for ZEVASKYN, the first FDA-approved therapy for treating RDEB wounds with a single application [1][2] - ZEVASKYN is a gene-modified cellular therapy developed through a collaboration between Abeona Therapeutics and Stanford Medicine, receiving FDA approval in April 2025 [1][2] - The treatment is now available at Lucile Packard Children's Hospital Stanford and Lurie Children's Hospital of Chicago, marking a significant milestone in its commercial launch [2] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being its flagship product for RDEB [7] - The company operates a fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [7] Product Details - ZEVASKYN (prademagene zamikeracel) is the first autologous cell sheet-based gene therapy for RDEB, addressing a severe skin disease caused by defects in the COL7A1 gene [3][4] - The therapy incorporates the COL7A1 gene into a patient's skin cells to produce functional type VII collagen, leading to clinically meaningful wound healing and pain reduction [3][4] Patient Support Initiatives - Abeona has established a comprehensive patient support program, Abeona Assist™, to help eligible patients access ZEVASKYN, including assistance with insurance benefits and logistical support [2]

Core Insights - Bio-Techne Corporation's Simple Western™ Technology was instrumental in the FDA approval of ZEVASKYN™, the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) developed by Abeona Therapeutics [1][5] - ZEVASKYN addresses a significant unmet medical need for RDEB patients, who previously had no durable therapies available [3][8] Company Overview - Bio-Techne Corporation is a global life sciences company that generated approximately $1.2 billion in net sales in fiscal 2024 and employs around 3,100 people worldwide [6] - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being its flagship product [8] Technology and Methodology - Simple Western was chosen by Abeona for its speed, reproducibility, sensitivity at picogram levels, and minimal sample volume requirements, effectively detecting both trimeric and monomeric forms of Collagen VII [2][4] - The platform provided GMP-compliant data to ensure consistency across ZEVASKYN product lots, overcoming challenges in analytical standardization and complex assay development [4] Clinical Significance - RDEB is a rare genetic skin disorder caused by mutations in the COL7A1 gene, leading to severe symptoms and increased risk of life-threatening complications [3] - The approval of ZEVASKYN marks a significant milestone in the field of cell-based gene therapy, providing a new treatment option for RDEB patients [5]