EGT-101

EGT-101 is a code name used for two distinct investigational therapeutic programs. The first is a clinical-stage biotherapeutic developed by Egret Therapeutics (a company founded by Turret Capital and experts from Stanford and Johns Hopkins) for the treatment of Delayed Cerebral Ischemia (DCI) following subarachnoid hemorrhage. The second is a gene therapy candidate developed by Esteve in collaboration with the Universitat Autònoma de Barcelona (UAB) for Sanfilippo A syndrome (Mucopolysaccharidosis type IIIA). The gene therapy version utilizes REGENX Biosciences' NAV rAAV9 vector technology to deliver a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to the central nervous system, addressing the underlying enzyme deficiency that leads to the accumulation of heparan sulfate in lysosomal storage disorders.