Abeona Therapeutics, Inc. is a clinical stage biopharmaceutical company, which engages in the development of gene therapy for severe and life-threatening rare diseases. It programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101 (AAV NAGLU), an AAV based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company was founded in 1974 and is headquartered in Cleveland, OH.
相关临床试验
16
8 进行中
药物批准
1
批准总数
监管机构
1
监管机构数
成立时间
N/A
进行中（未招募）
6
37.5%
已完成
5
31.3%
Enrolling By Invitation
1
6.3%
尚未招募
1
6.3%
招募中
1
6.3%
终止
2
12.5%
- The FDA granted standard full approval to Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) for pediatric mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome Type A. - Fayuvi is a one-time intravenous AAV9 gene therapy delivering a functional SGSH gene to restore the missing sulfamidase enzyme and reduce heparan sulfate accumulation. - Approval was supported by the pivotal Transpher A trial, in which treated patients scored 23.5 points higher on Bayley-III cognitive measures than an external natural history cohort (p<0.0001). - Ultragenyx expects commercial product to ship to Qualified Treatment Centers within 30-60 days, supported by its UltraCare program, and received a Priority Review Voucher.
- Abeona Therapeutics reports robust Q1 2026 results with revenue growth and EPS beat, supported by a 454.86% net income margin and 70.99% ROE. - The company is expanding treatment centers and advancing commercial progress with ZEVASKYN, its novel cell therapy product. - Analyst consensus remains favorable with 66.67% Buy ratings, though negative operating cash flow and a high P/S ratio of 31.73 present key risks. - Stock shows strong bullish momentum with moving averages signaling positive technical outlook, while RSI levels suggest potential near-term overbought conditions.
- The US House of Representatives unanimously passed the Mikaela Naylon Give Kids a Chance Act, reauthorizing the FDA's rare pediatric disease priority review voucher program that expired in December 2024. - The legislation combines regulatory frameworks to accelerate therapy access for children with rare diseases and certain cancers, with priority review vouchers currently valued at approximately $150 million. - Industry organizations and patient advocacy groups strongly support the bill, emphasizing its critical role in incentivizing pharmaceutical companies to develop treatments for rare pediatric conditions. - The bill now advances to the Senate for consideration before potentially reaching President Trump for final approval.
- Abeona Therapeutics has identified 30 eligible patients for ZEVASKYN gene therapy treatment at specialized centers, with 12 patients in the process of scheduling treatments for recessive dystrophic epidermolysis bullosa (RDEB). - Major commercial payers including UnitedHealthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans have published policies covering ZEVASKYN treatment. - The company plans to scale manufacturing capacity from current four to six monthly slots to ten slots by mid-2026, with further expansion planned by end of 2027. - ZEVASKYN received a permanent J-Code effective January 1, 2026, which is expected to streamline billing processes for clinics administering the therapy.
- ResearchAndMarkets.com has released a comprehensive report analyzing licensing deals in biotechnology from 2020 to 2025, providing insights into deal trends and structures. - The report covers hundreds of companies including major players like AbbVie, ACADIA Pharmaceuticals, and emerging biotech firms across various therapeutic areas and technology types. - Key features include financial terms analysis covering upfront payments, milestones, and royalties, plus access to actual contract documents for due diligence purposes. - The analysis enables benchmarking of transaction values and identification of the most active dealmakers in the biotechnology licensing landscape.
- Abeona Therapeutics has appointed James A. Gow, MD, MBA, MS, MHCM, as Senior Vice President, Head of Clinical Development & Medical Affairs, effective immediately. - Dr. Gow brings over 20 years of industry experience in clinical development and medical affairs, with recognized expertise in gene therapy, particularly in ophthalmology. - His clinical development track record includes leading programs from Phase 1 through post-marketing studies that resulted in FDA approvals of multiple drugs including Xibrom, Bromday, Prolensa, and Bepreve. - The appointment strengthens Abeona's leadership as the company advances its pipeline of cell and gene therapies, including ZEVASKYN for recessive dystrophic epidermolysis bullosa and AAV-based gene therapies for ophthalmic diseases.
- The FDA has selected Abeona Therapeutics' ABO-503 gene therapy for X-linked retinoschisis (XLRS) to participate in its highly competitive Rare Disease Endpoint Advancement (RDEA) Pilot Program. - The program provides enhanced communication and collaboration opportunities with the FDA to accelerate development and validation of novel efficacy endpoints for rare disease treatments. - ABO-503 has demonstrated preclinical efficacy in mouse models, showing structural and functional improvements including restored retinal architecture and improved visual function. - XLRS affects an estimated 35,000 patients in the United States and Europe combined, with no currently approved disease-modifying therapies available.
- Abeona Therapeutics received FDA approval in April for ZEVASKYN, the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder. - The company is investing $2.85 million to expand its Cleveland manufacturing facilities with a new 7,587-square-foot laboratory space to support commercial production and development of additional gene therapies. - Following the therapy approval, Abeona sold its Priority Review Voucher for $155 million, providing sufficient cash for over two years of operations and positioning the company for profitability by early 2026.
- AscellaHealth has partnered with Abeona Therapeutics to successfully commercialize ZEVASKYN, the first FDA-approved autologous cell sheet-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). - The collaboration resulted in AbeonaAssist, a customized patient support program designed to address the unique clinical, operational, and reimbursement challenges of this groundbreaking cell-based gene therapy. - Early results demonstrate enhanced patient compliance, retention, and satisfaction rates, highlighting the effectiveness of tailored support programs for complex gene therapies. - ZEVASKYN incorporates the functional COL7A1 gene into patients' own skin cells to produce type VII collagen, demonstrating clinically meaningful wound healing and pain reduction with a single surgical application.
- The FDA issued a Complete Response Letter for Ultragenyx's UX111 AAV gene therapy, citing chemistry, manufacturing and controls (CMC) observations that the company believes are readily addressable. - Clinical data were deemed robust by the FDA, with no issues noted regarding the clinical data package or clinical inspections, supporting the therapy's potential efficacy. - The regulatory setback delays potential approval to 2026, but Ultragenyx plans to resubmit the BLA after resolving manufacturing facility-related observations within the next few months. - UX111 represents a potential first treatment for Sanfilippo syndrome type A, a rare fatal lysosomal storage disease affecting 3,000 to 5,000 patients globally with a median life expectancy of 15 years.
