Abeona Therapeutics is developing cell and gene therapies for patients with serious diseases. Delivery of gene therapies to the desired tissue without causing an immune response is important for their success. Abeona is using their next-generation adeno-associated viruses (AAVs) to selectively deliver genetic payloads to the central nervous system, lungs, eyes, muscle, liver and other tissues. Abeona’s AAVs are non-replicating and have shown potential to evade immune responses generated by exposure to naturally-occuring AAV vectors. Abeona is applying their AAV technology for several diseases with therapies already in clinical trials. This includes EB-101, in Phase III trials for patients with Epidermolysis Bullosa and other undisclosed targets in preclinical phases.
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