May 21, 2026 —
Glafabra Therapeutics announced that the U.S. FDA has accepted its request for a face-to-face INTERACT meeting to discuss GT-GLA-S03, the company’s lead cell-based gene therapy candidate for Fabry disease. The meeting is scheduled for July 16, 2026, and is expected to help shape the company’s planned IND submission, targeted for the first quarter of 2027.
Fabry disease is a lysosomal storage disorder caused by deficiency of the alpha-galactosidase A enzyme, leading to accumulation of toxic lipid metabolites and progressive damage across multiple organs. Current standard treatment often relies on lifelong enzyme replacement therapy, requiring approximately 26 intravenous infusions per year. Over five years, that can mean around 130 clinic visits, creating a significant treatment burden for patients.
GT-GLA-S03 is designed to replace this lifelong infusion schedule with a single outpatient procedure intended to provide at least five years of therapeutic benefit. The therapy uses a patient’s own cells, which are genetically modified with a lentiviral vector to produce the missing enzyme. After reinfusion, the cells engraft in the bone marrow and generate white blood cells that circulate through the body and secrete the enzyme. Neighboring cells can then absorb the enzyme through cross-correction, helping reduce the toxic substrate accumulation that drives Fabry disease.
The program is supported by five-year human data from the FACTS trial, an investigator-initiated study conducted in Canada. In that study, five patients followed for five years had no product-attributable serious adverse events. Plasma lyso-Gb3, a key Fabry disease biomarker, decreased by 48% from the no-ERT baseline, and four of five patients maintained lyso-Gb3 below the elevated threshold at five years. Four of five patients also received their conditioning regimen as a same-day outpatient procedure.
Glafabra positions GT-GLA-S03 as a potentially broad option for Fabry patients regardless of GLA variant or antibody status. This may differentiate it from chaperone-based therapies, which are only suitable for certain variants, and from some other gene therapy approaches that may be limited by pre-existing anti-vector immunity. The company also notes that its reduced, non-myeloablative outpatient conditioning regimen using low-dose melphalan may be less burdensome than busulfan-based regimens.
Beyond Fabry disease, Glafabra is developing its Live-cel manufacturing platform for other lysosomal storage disorders, including GT-GAA-S04 for Pompe disease and GT-GBA1-S05 for Gaucher disease. The company sees the platform as potentially applicable across a broader set of enzyme deficiency disorders.
Overall, the FDA’s acceptance of the INTERACT meeting request represents an important early regulatory milestone for Glafabra. While GT-GLA-S03 still requires further regulatory review and clinical development, the program highlights continued innovation in cell-based gene therapy approaches designed to reduce lifelong treatment burden in rare metabolic diseases.
