Science

Our lead clinical program is EB-101, an autologous, engineered cell therapy currently in development for recessive dystrophic epidermolysis bullosa. Our development portfolio also features Ultragenyx-partnered ABO-102 (now UX111) for Sanfilippo syndrome type A (MPS IIIA), AAV-based gene therapies designed to treat ophthalmic and other diseases, next-generation AAV-based gene therapies using the novel AIM™ capsid platform, and internal AAV vector research programs.

Pipeline


EB-101

Recessive Dystrophic Epidermolysis Bullosa (RDEB)

EB-101 Current Phase
Phase III

Designations

  • Orphan Drug Designation (FDA)
  • Orphan Drug Designation (EMA)
  • Fast Track Designation (FDA)
  • Breakthrough Therapy Designation (FDA)
  • Regenerative Medicine Advanced Therapy Designation (FDA)

ABO-102 (UX111)

Sanfilippo Syndrome Type A (MPS IIIA)

ABO-102 (UX111) Current Phase
Phase I/II

Designations

  • 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-50X

Retinal Diseases

ABO-50X Current Phase
Preclinical

AIM™ Vectors

Undisclosed Targets

AIM™ Vectors Current Phase
Preclinical

EB-101 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

EB-101 is an autologous, engineered 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. 

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 1/2a clinical trial, EB-101 was well-tolerated and showed evidence of multi-year wound healing and pain reduction after treatment with a mean follow-up of 5.9 years and a maximum of eight years. 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.

The pivotal Phase 3 VIITAL™ study evaluated the efficacy, safety and tolerability of EB-101 in RDEB patients. The VIITAL™ study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. Learn more.

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 (UX111) for Sanfilippo Syndrome Type A (MPS IIIA)

ABO-102 (now UX111), partnered with Ultragenyx, 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.

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. 

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-50X for Retinal Diseases

Abeona is exploring the potential of ABO-50X for the treatment of monogenic eye disorders. ABO-50X is a group of AAV-based investigational products that were developed by Abeona researchers that leverage the next-generation AIMTM vector platform along with unique transgenes to tackle rare ocular diseases with high unmet need. Non-human primate data suggest that next-generation AIMTM AAV vectors can be used to efficiently target the retina, optic nerve, retinal pigmented epithelium and macula after direct ocular injection, creating the potential for new pipeline candidates that can address multiple eye disorders.

Manufacturing

Taking critical steps toward transitioning from clinical- to commercial-scale production and delivering cell and gene therapies to patients.

  • Producing EB-101 for Phase 3 VIITAL™ Study
  • Establishment of commercial-grade retrovirus manufacturing
  • Producing AAV-based gene therapies and vectors at scale
  • cGMP capabilities governed and validated by dedicated quality systems

The AIM™ Vector

Next-generation AAV capsids for use in gene therapies.

The AIM™ capsid library utilizes 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, potentially enabling the redosing of patients previously treated with AAV drug products.

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

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.