ABX-101

ABX-101 is a code name that has been applied to two distinct investigational products developed by different companies. 1. **Abeona Therapeutics (formerly PlasmaTech Biopharmaceuticals):** In this context, ABX-101 (also referred to as **ABO-101**) is an adeno-associated virus serotype 9 (AAV9)-based gene therapy developed for the treatment of **Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB; MPS IIIB)**. The therapy is designed to deliver a functional copy of the *NAGLU* gene to the central nervous system and peripheral tissues to address the deficiency of the N-acetyl-alpha-glucosaminidase enzyme, which leads to the toxic accumulation of heparan sulfate. 2. **Abalonex, LLC:** Separately, ABX-101 is an investigational drug developed for the treatment of **moderate-to-severe traumatic brain injury (TBI)**. It is administered via intramuscular injection and is being evaluated for its ability to improve clinical outcomes as measured by the Glasgow Outcome Scale Extended (GOS-E).