Latest news with #ZEVASKYN
Associated Press
3 days ago
- Business
- Associated Press
Abeona Therapeutics® to Present at the Jefferies Global Healthcare Conference
CLEVELAND, May 30, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that Vish Seshadri, Ph.D., Chief Executive Officer, and Madhav Vasanthavada, Ph.D., Chief Commercial Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, 2025 at 11:40 a.m. Eastern Time. A live webcast of the fireside chat can be accessed on the Investors section of the Abeona website under 'Events' at The webcast will be archived for 30 days. About Abeona Therapeutics 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 production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYN™, Abeona Assist™, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics [email protected]
Yahoo
3 days ago
- Business
- Yahoo
Abeona Therapeutics® to Present at the Jefferies Global Healthcare Conference
CLEVELAND, May 30, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that Vish Seshadri, Ph.D., Chief Executive Officer, and Madhav Vasanthavada, Ph.D., Chief Commercial Officer, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4, 2025 at 11:40 a.m. Eastern Time. A live webcast of the fireside chat can be accessed on the Investors section of the Abeona website under "Events" at The webcast will be archived for 30 days. About Abeona TherapeuticsAbeona 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 production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYN™, Abeona Assist™, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as 'may,' 'will,' 'believe,' 'anticipate,' 'expect,' 'intend,' 'potential,' and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, our ability to commercialize ZEVASKYN, the therapeutic potential of ZEVASKYN, whether the unmet need and market opportunity for ZEVASKYN are consistent with the Company's expectations, continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. CONTACT: Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@
Yahoo
12-05-2025
- Business
- Yahoo
Abeona Therapeutics® Enters into Agreement to Sell Priority Review Voucher for $155 Million
CLEVELAND, May 12, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it 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 the closing of the transaction. Abeona was awarded the PRV following the U.S. Food and Drug Administration (FDA) approval of ZEVASKYN™ (prademagene zamikeracel) on April 28, 2025. 'With proceeds from this PRV sale, we have sufficient cash for more than two years of operating expenses without the need for capital infusion and not accounting for ZEVASKYN sales,' said Joe Vazzano, Chief Financial Officer of Abeona. 'Furthermore, with ZEVASKYN becoming available to treat patients beginning third quarter of 2025, we anticipate becoming profitable in early 2026.' The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino (HSR) Antitrust Improvements Act. Stifel was lead financial advisor to Abeona on the transaction. Jefferies also served as financial advisor on the transaction. About Abeona Therapeutics 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 production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYNTM, Abeona AssistTM, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as 'may,' 'will,' 'believe,' 'anticipate,' 'expect,' 'intend,' 'potential,' and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, our ability to successfully generate commercial sales of ZEVASKYN and generate future revenue; the successful closing of our sale transaction for the Priority Review Voucher; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. CONTACT: Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@ in to access your portfolio
Forbes
30-04-2025
- Health
- Forbes
Breakthrough Gene Therapy For Butterfly Children's Disease
A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a landmark development for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN, the first autologous, cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a devastating skin condition often called "Butterfly Children's Disease." The approval marks a pivotal moment not only for the half-million people worldwide living with this rare condition but also demonstrates the power of an innovative venture philanthropy model that's reshaping how rare disease research is funded. The Wings of a Butterfly, the Resilience of a Warrior Children born with Epidermolysis Bullosa (EB) face extraordinary challenges from their first breath. Their skin, as fragile as a butterfly's wings, blisters and tears with the slightest friction, leading to open wounds, chronic pain, and susceptibility to infections. "For over 20 years, I've watched my son face the relentless challenges of EB with strength no child should ever have to summon," says Faye Dilgen, Board Member of EB Research Partnership (EBRP) and mother of John Hudson Dilgen, who lives with RDEB. "This new treatment doesn't just offer new relief, hope, and expanded options, it brings us closer to a future where children with EB won't have to endure the same struggles." For the approximately 500,000 people worldwide with EB, everyday activities that most take for granted—eating, sleeping, walking, and playing—become monumental tasks often requiring modification. In RDEB, which is one of the most severe forms of the condition, mutations in both copies of the COL7A1 gene prevent the body from producing functional Type VII collagen, a critical protein that anchors the dermis (inner layer) to the epidermis (outer layer) of the skin. The result is extremely fragile skin characterized by extensive blistering and severe wounds that can cover more than 30 percent of a patient's body surface—and in some cases up to 80 percent. These wounds cause debilitating pain and can remain open for years. Many that do close tend to reopen, leading to systemic complications that significantly impact both the length and quality of life. Gene therapy for rare pediatric disease getty A Scientific Breakthrough Delivered Through Persistence ZEVASKYN (pronounced "ZEE-vah-skin"), developed by Abeona Therapeutics, represents a scientific leap forward in the treatment of RDEB. Unlike previous treatments, ZEVASKYN addresses the genetic root cause of the disease by providing patients with their own skin cells (keratinocytes) that have been genetically modified to produce functional Type VII collagen. "Through a single surgical application, ZEVASKYN can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds," explains Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona Therapeutics. The results from clinical trials have been remarkable. In the pivotal Phase 3 VIITAL study, 81 percent of large chronic wounds treated with a single application of ZEVASKYN showed 50 percent or more healing after six months, compared to just 16 percent in control wounds treated with standard care. Even more promising, in earlier Phase 1/2a studies, researchers observed wound healing and pain reduction lasting for years after a single application. "In the completed Phase 1/2a study of ZEVASKYN, we have observed wound healing and pain reduction that have lasted for years after a single application," notes Jean Tang, M.D., Ph.D., professor of dermatology and lead principal investigator of the VIITAL study. "Today we can celebrate the availability of an exciting new therapeutic option made possible by the incredible courage of patients and families who participated in these clinical studies." The procedure involves surgically applying sheets of the patient's modified cells to wounded areas. In a single application, up to 12 credit card-sized sheets can be joined together to cover large areas or applied to multiple distinct wounds, allowing for significant coverage of affected body areas. A Revolutionary Funding Model for Rare Disease Research Perhaps equally remarkable as the scientific breakthrough is the innovative funding model that helped bring ZEVASKYN to market. EB Research Partnership (EBRP), the world's largest nonprofit funding research to accelerate treatments and find a cure for EB, invested in the groundbreaking work that led to ZEVASKYN at Stanford University under their pioneering Venture Philanthropy Model. This model, which has been highlighted for its leadership by Harvard Business School, Stanford Social Innovation Review, Yale University, the Milken Institute, and MIT, operates differently from traditional charitable giving. Rather than simply donating to research, EBRP invests in projects with commercial potential and negotiates for a share of the resulting financial returns, which are then reinvested into additional promising research. "EBRP is proud to be an early investor via our Venture Philanthropy Model in the science leading to ZEVASKYN at Stanford University," says Michael Hund, CEO of EBRP. "The collaboration between EBRP and Stanford exemplifies the mission of EBRP to advance commercially sustainable research aimed at treating and ultimately curing epidermolysis bullosa." The funding followed a highly competitive application and screening process overseen by EBRP's Scientific Advisory Board, composed of leading scientists and physicians. When the research showed promise, EBRP was able to realize a significant return on its investment and reinvest that capital back into additional promising EB research projects. This innovative approach addresses a critical gap in rare disease research funding. With over 10,000 rare diseases affecting approximately 400 million people worldwide, and 95% of these conditions having no approved treatments or cures, traditional funding models have proven insufficient. Venture philanthropy creates a sustainable cycle of investment and return that can accelerate research across multiple conditions simultaneously. "Abeona's development and advancement of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate the therapeutic landscape for not only EB, but many other rare diseases and conditions," Hund adds. Over the last decade, EBRP has made remarkable progress in its mission, raising over $70 million, funding more than 160 EB projects, and contributing to a 25-fold growth in the EB clinical trial landscape. Most importantly, the organization has directly funded two FDA-approved EB treatments, with ZEVASKYN marking the third FDA-approved treatment for the condition on the Horizon for Families For families living with RDEB, the approval of ZEVASKYN represents not just a new treatment option but a fundamental shift in their outlook on the future. The treatment is expected to be available beginning in the third quarter of 2025 through ZEVASKYN Qualified Treatment Centers (QTCs), strategically located across the U.S. to ensure nationwide access. Abeona has also established a comprehensive patient support program, Abeona Assist, offering personalized support for eligible patients and families throughout their treatment journey. "Having a new, uniquely differentiated, gene therapy for our patients with RDEB is a significant milestone in helping these special patients live fuller, pain-free and itch-free lives with less wounds," says Marissa Perman, MD, Section Chief of Dermatology and Director of the Epidermolysis Bullosa Multidisciplinary Clinic at Children's Hospital of Philadelphia. Hope for children with genetic disease getty A Model for the Future of Rare Disease Treatment The significance of ZEVASKYN's approval extends beyond its immediate impact on the EB community. It provides a blueprint for how innovative funding models, scientific breakthroughs, and patient advocacy can combine to overcome the challenges of rare disease treatment development. "This milestone is a testament to the dedication of scientists, researchers and medical professionals who have worked tirelessly to bring cutting edge treatments to those in need," says Joyce Teng, MD, PhD, professor in dermatology with multiple hospital affiliations. "It represents a scientific triumph, a profound step toward improving quality of life for individuals affected." As the third FDA-approved treatment for RDEB and the first autologous, cell-based gene therapy for the condition, ZEVASKYN represents a growing momentum in the field that offers hope not just to the EB community but to all those affected by rare diseases. "Public awareness of EB has never been stronger," notes EBRP, "and this most recent FDA approval underscores the momentum building around research, advocacy, and support." As the third FDA-approved treatment for RDEB and the first autologous, cell-based gene therapy for the condition, ZEVASKYN represents a growing momentum in the field that offers hope not just to the EB community but to all those affected by rare diseases. For the hundreds of thousands of "Butterfly Children" around the world, that momentum might finally mean wings strong enough to fly.
