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]

Company Overview - Senti Biosciences, Inc. is a clinical-stage biotechnology company focused on developing next-generation cell and gene therapies using its proprietary Gene Circuit platform [3] - The company aims to create new medicines that can precisely target and kill cancer cells while sparing healthy cells, enhancing specificity to target tissues, and allowing for control even after administration [3] Upcoming Event - Timothy Lu, M.D., Ph.D., Co-Founder and CEO of Senti Bio, will present at the LIVE! with Webull Corporate Connect: Virtual Biotech Investment Webinar on May 29, 2025, at 2:40 PM ET [1][2] Technology and Pipeline - Senti Bio's pipeline includes cell therapies engineered with Gene Circuits targeting challenging liquid and solid tumor indications [3] - The Gene Circuits have shown preclinical efficacy in both NK and T cells, with potential applications extending beyond oncology [3]

Core Points - Abeona Therapeutics Inc. has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million upon closing of the transaction [1] - The PRV was awarded following the FDA approval of ZEVASKYN™ (prademagene zamikeracel) on April 28, 2025 [1] - Proceeds from the PRV sale will provide sufficient cash for more than two years of operating expenses without the need for additional capital, excluding ZEVASKYN sales [2] - ZEVASKYN is expected to be available for patient treatment starting in the third quarter of 2025, with profitability anticipated in early 2026 [2] - The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act [2] 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 and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [4] - The company operates a fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio, which serves as the production site for ZEVASKYN [4] - Abeona's development portfolio includes adeno-associated virus (AAV)-based gene therapies targeting ophthalmic diseases with high unmet medical needs [4]

Core Viewpoint - Abeona Therapeutics is making significant progress with its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) aimed at treating recessive dystrophic epidermolysis bullosa (RDEB), with a target action date set for April 29, 2025 [1][6]. Financial Results - For the full year 2024, Abeona reported a net loss of $63.7 million, or $1.55 loss per common share, compared to a net loss of $54.2 million, or $2.53 loss per common share in 2023 [7][11]. - Research and development expenses increased to $34.4 million in 2024 from $31.1 million in 2023, primarily due to increased headcount for manufacturing capacity expansion [7][11]. - General and administrative expenses rose to $29.9 million in 2024 from $19.0 million in 2023, attributed to costs associated with commercial launch preparations [7][11]. Cash Position and Runway - As of December 31, 2024, the company had cash, cash equivalents, short-term investments, and restricted cash totaling $98.1 million, up from $52.6 million a year earlier [4][5]. - Abeona estimates that its current financial resources are sufficient to fund operations into 2026, not accounting for potential revenue from pz-cel sales or proceeds from a Priority Review Voucher (PRV) [5]. Regulatory and Development Updates - The FDA has accepted the BLA resubmission for pz-cel and discussions are ongoing regarding post-marketing requirements and the draft label [2][6]. - Abeona plans to treat its first patient with pz-cel in the third quarter of 2025, pending FDA approval [1][6]. - The company is actively preparing for commercialization, including onboarding treatment centers and engaging payers to ensure patient access [6]. Intellectual Property - Abeona has obtained two additional patents for pz-cel, extending patent protection for its use in treating RDEB to June 2037 and for its packaging and transport system to July 2040 [6]. Conference Call - A conference call to discuss financial results and company updates is scheduled for March 20, 2025, at 8:30 a.m. ET [8].