Update: April 2021
This Phase 1/2 trial has now fully enrolled and is not accepting additional patients. We are hopeful that a Phase 3 trial will be initiated in the future but no timeline is currently available.
EGT-101 is Esteve’s potential first-in-class AAV-9 gene therapy that is administered via a one-time intracerebroventricular injection into the cerebrospinal fluid.
EGT-101 consists of an AAV9 vector containing the human sulfamidase (SGSH) transgene and it aims to restore SGSH functional deficiency in patients with Sanfilippo Syndrome Type IIIA.
Information is available on Esteve’s website.