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 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.
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- EB-101 Current Phase
- Phase III
- Orphan Drug Designation (FDA)
- Orphan Drug Designation (EMA)
- Breakthrough Therapy Designation (FDA)
- Regenerative Medicine Advanced Therapy Designation (FDA)
- ABO-50X Current Phase
- AIM™ Vectors Current Phase
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-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.
ABO-504 for Stargardt Disease
Abeona’s internal research and development team developed ABO-504, which is designed to efficiently reconstitute the full-length ABCA4 gene by implementing a dual AAV vector strategy using the Cre-LoxP recombinase system. In May 2021 at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Abeona reported preclinical data demonstrating the ability of the dual AAV vector system to produce full length ABCA4 protein in cell culture. Recent proof-of-concept studies have extended these findings by showing expression of ABCA4 mRNA and full-length ABCA4 protein in the retina of subretinally dosed abca4-/- knockout mice, at levels similar to endogenous ABCA4 in wild-type animals.
ABO-503 for X-linked Retinoschisis (XLRS)
ABO-503, composed of a functional human RS1 packaged in the novel AIM™ capsid AAV204, has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies have demonstrated robust RS1 expression in the retina, improved cone photoreceptor density and overall photoreceptor cell survival, as well as a restoration of outer retina architecture.
ABO-505 for Autosomal Dominant Optic Atrophy (ADOA)
ABO-505 is designed to express a functional copy of human Opa1 in the retina following para-retinal injection. AB0-505 aims to take advantage of the robust optic nerve and retinal ganglion cell (RGC) transduction ability of AAV204 to deliver its genetic payload to the cells most affected by ADOA. Preclinical studies have confirmed expression of Opa1 in both cell culture and the retinas of dosed wild-type and disease model animals. Initial efficacy results suggest an improvement in retinal signaling to the brain, and improved visual acuity in treated mutant mice.
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
- 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.