Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I).
The SB-318 IND application is now active and enables Sangamo to initiate a Phase 1/2 clinical study (SB-318-1502) designed to assess the safety, tolerability and potential efficacy of SB-318 in adults with varying severities of MPS I.
SB-318 is Sangamo's second in vivo genome editing application cleared by the FDA; the first being for hemophilia B (SB-FIX). Both programs are based on Sangamo's proprietary In Vivo Protein Replacement Platform (IVPRP™), a single treatment strategy designed to produce stable circulating levels of a therapeutic protein from a patient's liver for the lifetime of the individual.
SB-318 treatment is intended to eliminate the need for enzyme replacement therapy (ERT) which is the current standard of care for the majority of patients with MPS I. ERT for MPS I often requires weekly infusions of a recombinant form of the enzyme alpha-L-iduronidase (IDUA) which is missing, or defective, in patients with the disorder. While the infusions take several hours, circulating levels of IDUA are undetectable within hours of the treatment due to the replacement protein's short half-life.
