Latest news with #AbeonaTherapeutics
Medscape
04-07-2025
- Health
- Medscape
RDEB: Large Wounds Healed With Genetically Corrected Grafts
In a recently published phase 3 trial, credit card-sized cultured skin grafts corrected for the COL7A1 mutation that causes recessive dystrophic epidermolysis bullosa (RDEB) and enabled most patients to achieve at least 50% reductions in the size of large chronic wounds, with an overall mean pain score reduction of more than 2 points at week 24. In April 2025, prademagene zamikeracel (Zevaskyn, Abeona Therapeutics) became the first FDA-approved cell-based genetic therapy when it was approved for the treatment of wounds in adult and pediatric patients with RDEB. It is the first commercially available RDEB treatment to demonstrate sustained wound healing and pain reduction for large, chronic RDEB wounds, according to investigators. 'These wounds are the most terrible and difficult to treat in our patients,' the study's lead principal investigator Jean Y. Tang, MD , PhD, professor of dermatology at Stanford University School of Medicine in Stanford, California, said in an interview. 'To have a therapy using the patient's cells to suture on, hopefully close their wounds, and reduce their pain is monumental.' Jean Y. Tang, MD , PhD For the VIITAL trial, published online on June 23 in The Lancet , Tang and colleagues enrolled 11 patients with clinically and genetically confirmed RDEB (median age, 21 years) and no evidence of immune response to type VII collagen. To reduce the likelihood of immunogenicity, only patients with the amino-terminal NC1 fragment of type VII collagen could enroll. Investigators selected 43 wounds of at least 6 months' duration measuring at least 20 cm2 for treatment and compared these results against standard care for 43 randomly assigned control wounds matched for size, chronicity, and location. Grafting Process Using 8-mm punch biopsies from unaffected skin, investigators transduced isolated keratinocytes with a retrovirus carrying the full-length human COL7A1 gene, then used those keratinocytes to culture up to 12 40 cm2 sheets of autologous keratinocytes per patient. After 25 days, surgeons sutured up to six sheets of prademagene zamikeracel per patient, with each procedure taking 3-4 hours. To minimize pressure and friction, patients remained hospitalized with no changes of nonadhesive contact dressings for 7 days postsurgery. Images of wounds randomly assigned to prademagene zamikeracel or control at baseline, surgery, and week 24. Investigator assessments showed that 24 weeks posttreatment, 81% of treated patients achieved at least 50% healing from baseline vs 16% of control wounds ( P < .0001). Mean pain reduction from baseline (measured with the Wong-Baker Faces scale within 3 hours after dressing change) was 3.07 among treated patients vs 0.90 for control wounds ( P = .0002). Also at week 24, 16% of treated wounds achieved complete healing, with a 2.0-point decrease in itch severity from baseline. The corresponding figures for control wounds were 0 (healing) and 0.5 (itch). In the past 3 years, the FDA and the European Medicines Agency also have approved topical beremagene geperpavec (Vyjuvek) and birch triterpenes (Filsuvez) for dystrophic EB. However, wrote Tang and colleagues, the wounds treated with these therapies were mostly less than 20 cm2, and both treatments require repeated application. Nor did they improve pain or itchin clinical trials, added Tang. Having the first permanent gene correction for RDEB is very exciting, said Amy Paller, MS, MD, professor and chair of Dermatology and professor of pediatrics at Northwestern University, Chicago. She was not involved with the phase 3 study but will run the first of several specialized centers where prademagene zamikeracel will be applied. Amy Paller, MS, MD 'This is the first instance in our field where a gene has been corrected for grafting and is commercially available,' Paller said. 'It's something that we and our patients dreamed about for genetic skin disorders.' Logistics and Labor Performing the treatment is logistically complex and 'incredibly labor-intensive,' Paller said. The process requires rushing biopsies to Abeona's good manufacturing practice facility in Cleveland, where over the next few weeks, the keratinocytes are grown out, corrected, expanded markedly, and quality tested. 'It's a very expensive procedure with many moving parts,' she said. Accordingly, Paller plans to start with three patients from her own practice, beginning in August. Additionally, she is consulting with other interested families in the Midwest and will soon expand outreach to her other patients. 'I want experience with the process in patients I have known for years before grafting additional patients,' she explained. Prademagene zamikeracel's retroviral component may provoke discussion. Tang explained, 'We take the biopsy from the patient's skin, grow their keratinocyte skin cells, and use a retrovirus containing wild-type collagen VII to introduce that into the patient's skin cells. There's always a theoretical concern of retroviruses maybe hitting off-target genes, but so far, we and others haven't seen that.' In a phase 1/2a study, investigators followed seven patients treated with what was then known as EB-101 for a mean of 5.9 years. There were no serious adverse events related to treatment, with no gene therapy-related cutaneous or extracutaneous malignancies or evidence of systemic replication-competent retrovirus infections in serum samples from patients. The beauty of grafting skin, Paller added, is that development of a tumor — while unexpected — would be easily visible and biopsied, just as dermatologists now biopsy for suspected squamous cell carcinoma, a feared complication related to the scarred skin in patients with RDEB. Treated patients will require a long-term commitment to surveillance, she said, with a low threshold for considering biopsy if a change suggesting carcinoma is seen. The FDA recommends that manufacturers of genetic products follow patients for 15 years posttreatment. Clinical and Research Implications Although the phase 3 study showed the utility of correcting genetically defective collagen VII in treating RDEB, said Tang, the cell therapy approach could prove useful for additional genetic skin diseases such as ichthyosis and Gorlin syndrome. Paller said she hopes that junctional EB will be the next candidate for gene-corrected grafts. However, she added, with more extensive clinical experience and cost reductions over time, grafting of gene-corrected skin could be considered to improve focal areas in other forms of EB and genetic skin disorders. For the near term, Paller said she also hopes that insurers will not block access to the other approved RDEB treatments for patients who undergo prademagene zamikeracel treatment. 'I trust that that won't happen because these patients are so needy,' she said. To help patients access treatment, Abeona offers the Abeona Assist program, which helps patients understand their insurance benefits and financial assistance options and provides travel and logistical assistance. 'As far as I'm concerned,' said Paller, 'each patient with EB should have everything at our disposal to help — this is such a horrible disease. If I can graft a 12 credit card-sized area and then keep them going with tricks for other areas, I'll be very happy.' The study was funded by Abeona Therapeutics, which developed prademagene zamikeracel, which also conducted data analysis and employs several study co-authors. Tang is listed on the prademagene zamikeracel patent, which is licensed by Stanford University to Abeona, but she receives no royalties. Additionally, Tang has consulted on EB-related therapeutics for BridgeBio and Fibroderm. Paller served on the VIITAL data safety monitoring board and has consulted for Chiesi, Krystal, and Castle Creek Biosciences.
Yahoo
03-07-2025
- Business
- Yahoo
Abeona Therapeutics® Announces Option Exercise by Beacon Therapeutics for Novel AAV204 Capsid for Ophthalmology Gene Therapy
Abeona will receive a license payment and potential development, regulatory, and sales milestones, and royalties CLEVELAND, July 01, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that Beacon Therapeutics, an ophthalmic gene therapy company and Syncona portfolio company, has exercised its option to license Abeona's patented AAV204 capsid for use in potential gene therapies for a range of prevalent and rare retinal diseases that result in blindness. This worldwide, non-exclusive license is pursuant to the agreement between Abeona and Beacon, announced in July 2024, to evaluate the therapeutic potential of AAV204. AAV204, a novel AAV capsid from the AIM™ capsid library licensed by Abeona from the University of North Carolina at Chapel Hill, has been shown to achieve high macular and optic nerve transduction levels after para-retinal administration and has also been shown to facilitate transduction of both the inner and outer retina after intravitreal administration in mice and non-human primates. 'Beacon's option exercise further validates AAV204's potential to enable targeted delivery of gene therapies in rare and prevalent ophthalmic diseases,' said Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development at Abeona Therapeutics. 'Our non-exclusive agreement with Beacon enables us to fully explore the therapeutic value of AAV204 in additional ophthalmic diseases.' The exercise of this license option concludes Beacon's initial evaluation of the AAV204 capsid for development and commercialization of gene therapies and gives Beacon the right to use AAV204 in connection with up to five gene or ophthalmology disease targets. Under the terms of the agreement, Abeona will receive an undisclosed upfront license payment with additional payments upon the achievement of certain development, regulatory, and sales milestones, along with tiered royalties on worldwide net sales for licensed products incorporating AAV204. About the AIM™ capsid libraryThe AIM™ capsid library is a collection of novel AAV serotypes that target delivery of genetic payloads to key tissues implicated in devastating genetic diseases, including the central nervous system (including the retina), lungs, eye, muscle, liver and other tissues, with potentially improved tropism profiles. AIM™ vectors have shown the potential to evade the immune response generated by exposure to naturally-occurring AAV vectors in preclinical studies. AAV204 is covered by U.S. Patent Nos. 10,532,110 and 10,561,743. 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@ in to access your portfolio
Yahoo
01-07-2025
- Business
- Yahoo
Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
CLEVELAND, July 01, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has granted equity awards to new non-executive employees who joined the Company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4). On June 30, 2025, the Compensation Committee of Abeona's Board of Directors granted restricted stock equity awards as a material inducement to employment to 21 individuals hired by Abeona, which equity awards relate to, in the aggregate, up to 48,715 restricted shares of Abeona common stock. One-third of the shares subject to such restricted stock awards will vest yearly on each anniversary of the Grant Date, such that the shares subject to such restricted stock awards granted to each employee will be fully vested on the third anniversary of the Grant Date, in each case, subject to each employee's continued employment with Abeona on the applicable vesting dates. 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 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, the timing and outcome of the FDA's review of our BLA resubmission for pz-cel; the FDA's grant of a Priority Review Voucher upon pz-cel approval; 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 retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
02-06-2025
- Business
- Associated Press
Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
CLEVELAND, June 02, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has granted equity awards to new non-executive employees who joined the Company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4). On May 31, 2025, the Compensation Committee of Abeona's Board of Directors granted restricted stock equity awards as a material inducement to employment to five individuals hired by Abeona, which equity awards relate to, in the aggregate, up to 11,500 restricted shares of Abeona common stock. One-third of the shares subject to such restricted stock awards will vest yearly on each anniversary of the Grant Date, such that the shares subject to such restricted stock awards granted to each employee will be fully vested on the third anniversary of the Grant Date, in each case, subject to each employee's continued employment with Abeona on the applicable vesting dates. 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 Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics [email protected]
Associated Press
30-05-2025
- 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]
