Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- pz-cel (prademagene zamikeracel) Current Phase
- ABO-504 Current Phase
X-Linked Retinoschisis (XLRS)
- ABO-503 Current Phase
Autosomal Dominant Optic Atrophy (ADOA)
- ABO-505 Current Phase
Sanfilippo Syndrome Type A (MPS IIIA)
- UX111* Current Phase
- Phase 1/2
- TSHA-102** Current Phase
- Phase 1/2
Infantile Batten Disease (CLN1 Disease)
- TSHA-118** Current Phase
- Phase 1/2
pz-cel (prademagene zamikeracel) for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
pz-cel (prademagene zamikeracel) 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 pz-cel (prademagene zamikeracel) 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.
pz-cel (prademagene zamikeracel) 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, pz-cel (prademagene zamikeracel) 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 pz-cel (prademagene zamikeracel) 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. pz-cel (prademagene zamikeracel) 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 pz-cel (prademagene zamikeracel) 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.
Taking critical steps toward transitioning from clinical- to commercial-scale production and delivering cell and gene therapies to patients.
- Producing pz-cel (prademagene zamikeracel) 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.