Our development portfolio 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.
ABO-503
X-Linked Retinoschisis (XLRS)
- ABO-503 Current Phase
- Preclinical
ABO-504
Stargardt Disease
- ABO-504 Current Phase
- Preclinical
ABO-505
Autosomal Dominant Optic Atrophy (ADOA)
- ABO-505 Current Phase
- Preclinical
UX111*
Sanfilippo Syndrome Type A (MPS IIIA)
- UX111* Current Phase
- Phase 1/2
TSHA-102**
Rett Syndrome
- TSHA-102** Current Phase
- Phase 1/2
TSHA-118**
Infantile Batten Disease (CLN1 Disease)
- TSHA-118** Current Phase
- Phase 1/2
Manufacturing
Taking critical steps toward transitioning from clinical- to commercial-scale production and delivering cell and gene therapies to patients.
- Conducted manufacturing of retroviral vector and drug product 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.