Careers Contact Us Facebook Twitter LinkedIn Home About Science Clinical Trials Patients & Families Investors & Media Science PIPELINE MANUFACTURING THE AIMTM VECTOR Pipeline PRECLINICAL PHASE I/II PHASE III DESIGNATIONS EB-101 Recessive Dystrophic Epidermolysis Bullosa (RDEB) View Clinical Trial Orphan Drug Designation (FDA) Orphan Drug Designation (EMA) Fast Track Designation (FDA) Breakthrough Therapy Designation (FDA) Regenerative Medicine Advanced Therapy Designation (FDA) ABO-102 Sanfilippo Syndrome Type A (MPS IIIA) View Clinical Trial Orphan Drug Designation (FDA) Orphan Drug Designation (EMA) Rare Pediatric Disease Designation (FDA) Fast Track Designation (FDA) Regenerative Medicine Advanced Therapy Designation (FDA) Priority Medicines (EMA) ABO-101 Sanfilippo Syndrome Type B (MPS IIIB) View Clinical Trial Orphan Drug Designation (FDA) Orphan Drug Designation (EMA) Rare Pediatric Disease Designation (FDA) Fast Track Designation (FDA) AB0-201 Juvenile Batten Disease (CLN3) Orphan Drug Designation (FDA) Orphan Drug Designation (EMA) AIM™ Vectors Undisclosed Targets ABO-401 Cystic Fibrosis (CF) ABO-50X Retinal Diseases EB-101 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) EB-101 is an autologous, gene-corrected cell therapy for RDEB, a rare connective tissue disorder without an approved treatment in which patients suffer with severe epidermal wounds that impact the length and quality of their lives. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce Type VII collagen that helps anchor the dermal and epidermal layers of the skin. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into an RDEB patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and skin function. Investigators are currently enrolling the VIITAL™ Study, a pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of RDEB patients. Learn more. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating. In a Phase 1/2 clinical trial, EB-101 was well-tolerated and resulted in significant and durable wound healing, with up to 5 years of follow-up. Continuous Type VII collagen expression was observed more than two years after treatment, and no drug-related serious adverse events have been observed to date. Abeona holds several regulatory designations in the U.S. and EU for EB-101 that allow increased interactions and guidance from the FDA and EMA. In the U.S., these include Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric Disease designations and Orphan Drug designation in both the U.S. and EU. ABO-102 for Sanfilippo Syndrome Type A (MPS IIIA) ABO-102 is a novel, one-time gene therapy aimed at reversing the effects of the genetic error that causes Sanfilippo syndrome, or MPS IIIA, a rare disease without an approved treatment that is characterized by rapid neurodevelopmental and physical decline. People with MPS IIIA have a defect in the SGSH gene that hinders the breakdown of toxic sugars called glycosaminoglycans (GAGs) that accumulate throughout the body. This accumulation causes progressive cell damage that can slow development and cause other autism-like symptoms. ABO-102 is designed to deliver a working copy of the defective SGSH gene to cells of the central nervous system and peripheral organs using an AAV vector platform. This one-time IV infusion may help the body produce an enzyme to break down GAGs. We are conducting a pair of ongoing Phase I/II clinical trials evaluating ABO-102 as a treatment for MPS IIIA: The Transpher A study is an open-label, dose-escalation clinical trial assessing ABO-102 for younger, higher-functioning patients who are likely to receive the most benefit from treatment. Preliminary results from the study demonstrate that ABO-102 has been well tolerated to date with preservation of neurocognitive development 18-24 months post treatment among the youngest patients in the highest dose cohort. Robust and sustained improvement observed in biomarkers confers additional evidence of a clear biological effect following ABO-102 administration. Study ABT-003 is an open-label clinical trial assessing the safety of ABO-102 for patients with middle and advanced phases of the disease. The study is planned to enroll up to 12 patients across three global study sites. Eligible patients must have a cognitive development quotient lower than 60 and must be ambulatory, with or without assistance. Abeona holds several regulatory designations in the U.S. and EU for ABO-102 that allow increased interactions and guidance from the FDA and EMA. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations for the program. ABO-101 for Sanfilippo Syndrome Type B (MPS IIIB) ABO-101 is a novel, one-time gene therapy aimed at reversing the effects of the genetic error that causes Sanfilippo syndrome type B, or MPS IIIB, a rare disease without an approved treatment that is characterized by rapid neurodevelopmental and physical decline. People with MPS IIIB have a defect in the NAGLU gene that hinders the breakdown of toxic sugars called glycosaminoglycans (GAGs) that accumulate throughout the body. This accumulation causes progressive cell damage that can slow development and cause other autism-like symptoms. ABO-101 is designed to deliver a working copy of the defective NAGLU gene to cells of the central nervous system and peripheral organs using an AAV vector platform. This one-time IV infusion may help the body produce an enzyme to break down GAGs. We are enrolling eligible patients with MPS IIIB into an ongoing Phase 1/2 clinical trial of ABO-102. The Transpher B study, is an open-label, dose-escalation clinical trial evaluating ABO-101 as a treatment for MPS IIIB. Interim results from the study demonstrate that ABO-101 has been well tolerated to date and shown to improve multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101, and Orphan Drug designation in both the U.S. and EU. AB0-201 for Juvenile Batten Disease (CLN3) ABO-201 is a novel, one-time gene therapy for the treatment of juvenile Batten disease, or CLN3 disease, a rare disease without an approved treatment in which patients face precipitous developmental regression. The first noticeable sign of all forms of Batten disease is often progressive vision impairment that can end in blindness. Many children also lose the ability to speak in complete sentences and to walk or sit. People with the disorder have a defect in the CLN3 gene, which is synonymous with a deficiency in proteins critical to lysosomal function that lead to abnormal buildup of lipopigments, and result in neuroinflammation and neurodegeneration. ABO-201 is designed to deliver a working copy of the defective CLN3 gene using an AAV vector platform. Pre-clinical data suggest that ABO-201 may result in significant, dose-dependent biodistribution into the central nervous system and peripheral organs in adult mice with CLN3 disease. Abeona holds Orphan Drug designation for ABO-201 in the U.S. and EU. ABO-401 for Cystic Fibrosis (CF) ABO-401 is an investigational gene therapy for the treatment of cystic fibrosis (CF), and the first therapeutic candidate born from the next-generation AIM™ vector platform. Pre-clinical data suggest that ABO-401 has the potential to address all mutations of CF, including the most common mutation, delta-F508. Additional pre-clinical data suggest that ABO-401 efficiently targets lung cells and corrects the underlying CF chloride channel deficit, regardless of underlying mutations of the CF transmembrane conductance regulators. ABO-50X for Retinal Diseases Abeona is exploring the potential of ABO-50X for the treatment of genetic eye disorders. ABO-50X was discovered by Abeona researchers and was born from the next-generation AIMTM vector platform. Non-human primate data suggest that next-generation AIMTM AAV vectors can be used to efficiently target the retinal epithelium after intravitreal injection, creating the potential for new pipeline candidates that can address multiple eye disorders. Additional pre-clinical data show that certain AIMTM capsids that demonstrated high tropisms for central nervous system tissue can evade neutralizing antibodies against naturally occurring AAV serotype, and potentially enable redosing in patients that have previously received an AAV injection. Your browser doesn't support HTML5 video. Here is a link to the video instead. Manufacturing Taking critical steps toward transitioning from clinical- to commercial-scale production and delivering gene and cell therapies to patients. Ready to produce EB-101 for Phase 3 VIITAL™™ Study™ Producing AAV-based gene therapies and vectors CMC capabilities governed & validated by dedicated quality systems The AIM™ Vector Next-generation AAV capsids for use in gene therapies. The AIM™ capsid library can utilize AAV biology to selectively target delivery of genetic payloads to the central nervous system, lungs, eye, muscle, liver and other tissues. AIM™ vectors are non-replicating and have shown the potential to evade the immune responses generated by exposure to naturally-occurring AAV vectors. The Abeona AIM™ capsid library: Selective tissue targeting Non-replicating Multiple routes of delivery Potential to re-dose patients previously-treated with AAV delivered gene therapy 100+ capsids The safety and efficacy of the investigational use of these products has not been determined. There is no guarantee that the investigational uses listed will be filed with and/or approved for marketing by a regulatory agency. © 2021 Abeona Therapeutics Inc. All Rights Reserved. | Privacy Policy