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]

Financial Performance - Abeona Therapeutics Inc. closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million on June 27, 2025, contributing to a total cash resource of approximately $225 million as of June 30, 2025 [1][2] - The proceeds from the PRV, combined with existing cash, provide the company with over two years of operating capital, ensuring financial flexibility without the need for further capital infusion [2] Product Development - ZEVASKYN™ (prademagene zamikeracel) is the first and only U.S. approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [2][3] - The company anticipates the first patient treatment with ZEVASKYN in Q3 2025, with profitability projected for early 2026 [2] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which serves as the production site for ZEVASKYN [3]

Core Insights - Abeona Therapeutics has announced that Beacon Therapeutics has exercised its option to license Abeona's patented AAV204 capsid for potential gene therapies targeting various retinal diseases, which could lead to significant financial benefits for Abeona through license payments and royalties [1][3]. Group 1: Licensing Agreement - The licensing agreement with Beacon Therapeutics is worldwide and non-exclusive, allowing Beacon to develop gene therapies for up to five ophthalmic disease targets using AAV204 [1][3]. - Abeona will receive an undisclosed upfront license payment, along with additional payments tied to development, regulatory, and sales milestones, plus tiered royalties on worldwide net sales of products incorporating AAV204 [3]. Group 2: AAV204 Capsid Details - AAV204 is a novel AAV capsid from the AIM™ capsid library, which has demonstrated high transduction levels in the macula and optic nerve after administration in preclinical studies [2][4]. - The AIM™ capsid library includes various AAV serotypes designed to improve delivery to tissues affected by genetic diseases, potentially evading immune responses [4]. Group 3: Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including its autologous cell-based gene therapy ZEVASKYN™ for treating recessive dystrophic epidermolysis bullosa [5]. - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which supports the commercial production of ZEVASKYN [5].

Core Insights - Abeona Therapeutics Inc. has been added to the U.S. small-cap Russell 2000® Index and the broad-market Russell 3000® Index, effective June 30, 2025, marking a significant milestone for the company [1][2] - The inclusion in these indexes is expected to enhance the company's visibility and attract a wider range of investors, particularly following the FDA approval of ZEVASKYN™ [2] - The Russell 3000® Index includes the 3,000 largest U.S.-traded stocks, and membership is updated annually, providing a benchmark for investment strategies [3] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [4] - ZEVASKYN™ is the first autologous cell-based gene therapy approved for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [4] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is responsible for ZEVASKYN's commercial production [4]

Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company's flagship product, ZEVASKYN™ (prademagene zamikeracel), is the first and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [3] - Abeona operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is dedicated to the commercial production of ZEVASKYN [3] Upcoming Events - The CEO, Vish Seshadri, and the Chief Commercial Officer, Madhav Vasanthavada, will participate in a fireside chat at the Jefferies Global Healthcare Conference on June 4, 2025, at 11:40 a.m. Eastern Time [1] - A live webcast of the event will be available on the Investors section of the Abeona website and will be archived for 30 days [2] Product Development - Abeona's development portfolio includes adeno-associated virus (AAV)-based gene therapies targeting ophthalmic diseases with significant unmet medical needs [3] - The company is evaluating next-generation AAV capsids to enhance tropism profiles for various severe diseases [3]

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][5] - The company is transitioning to a commercial-stage entity with the launch of ZEVASKYN and has activated its first treatment center [2][4] Recent Developments - ZEVASKYN's FDA approval marks a significant milestone for RDEB patients, with the first treatment expected in Q3 2025 [5] - Abeona has secured a $155 million non-dilutive capital through the sale of its Rare Pediatric Disease Priority Review Voucher (PRV), which will fund operations for over two years [3] - The company has entered into value-based agreements with commercial payer groups, ensuring access to ZEVASKYN for approximately 100 million commercially-insured lives [5] Financial Results - As of March 31, 2025, the company reported cash and cash equivalents totaling $84.5 million, down from $98.1 million at the end of 2024 [6] - Research and development expenses increased to $9.9 million in Q1 2025 from $7.2 million in Q1 2024, primarily due to scaling up manufacturing for ZEVASKYN [7] - The net loss for Q1 2025 was $12.0 million, a significant reduction from a net loss of $31.6 million in Q1 2024 [8][14]

Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell- based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company's fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial produ ...