Posted by Michael Wonder on 07 Apr 2019
Abeona Therapeutics receives FDA fast track designation for ABO-101 for treatment of Sanfilippo syndrome type B
4 April 2019 - Ongoing Phase 1/2 study of ABO-101 enrolling eligible patients with MPS IIIB.
Abeona Therapeutics Inc today announced that the U.S. FDA has granted fast track designation to ABO-101, the Company’s novel one-time gene therapy for Sanfilippo syndrome type B (MPS IIIB).
ABO-101 is designed to deliver a functional copy of the NAGLU gene to the central nervous system and peripheral tissues. Abeona is enrolling eligible patients with MPS IIIB at sites in the U.S. and Spain and expects to activate additional sites globally this year to accelerate enrolment.
