Abeona Therapeutics Inc (NASDAQ: ABEO) is a fully integrated authority in the cell and gene therapy space. It recently announced that it had a fruitful Type-B meeting with the FDA (Food and Drug Administration) regarding the approval and support trial of its developmental-stage AAV-based ABO-102 gene therapy. This medication is designed to treat people suffering from Type-A Sanfilippo syndrome (MPS IIIA). The ABO-102 treatment is currently being analyzed and examined in the Transfer-A single-arm clinical study in people suffering from MPS IIIA.
The Transfer-A clinical study will act as the primary research study for this AAV-based gene therapy based on the FDA Type-B meeting. In fact, depending on the information collected, this therapy has the potential to support a BLA (Biologics License Application) submission. Additionally, the company also agreed with the FDA on the study’s primary endpoints. Finally, the company plans to work closely with the US FDA via the RMAT (regenerative medicine advanced therapy) to develop a strong data package for ABO-102 registration.
Abeona’s Chief Executive Officer, Michael Amoroso, said that they greatly appreciate both the collaborative exchange and guidance provided by the FDA concerning the pivotal clinical trial to support the approval of this AAV-based ABO-102 gene therapy for patients with MPS IIIA. Especially for those who don’t have access to any approved treatments. The CEO went on to say that from 2016 until now, they’ve managed to treat twenty-one individuals in the Transfer-A clinical trial. He said they were delighted about the magnitude of benefit and safety seen in their investigational treatment in the younger kids from the high dosage categories reported at the beginning of the year.
The patients the company is looking to treat have huge unmet requirements. Abeona remains totally focused on excelling with their operations by offering two potentially pivotal information packages; one for EB-101 and ABO-102.