
Pipeline Overview
The programs at Glafabra Therapeutics target a variety of metabolic disorders with a novel, long-lasting cell therapy. We are bringing two types of cell-based gene therapies to patients as durable solutions to increase the freedom in the lives of those suffering from debilitating metabolic disorders.
STEM Therapy (Clinical Stage)
Lead Therapy - Fabry Disease:
Our STEM therapy approach is an improvement over intermittent enzyme exposure with recombinant protein enzyme replacement therapy (ERT) . Unlike traditional ERT, our STEM therapy provides uninterrupted cross-correction therapy - the patient obtains a more continuous supply of enzyme. The durability of our STEM therapy is now demonstrated to exceed 5 years ((PMID 39794302). This indicates the hassle of every other week infusions can be avoided for many years. Furthermore, the transplanted leukocytes are known to spread systemically to a wide variety of compartments in the body, including the brain, thus the blood brain barrier problem with traditional recombinant protein transfusions is a much lower obstacle. As a result, successful treatment of neurological complications of enzyme deficiency can be expected.
TRAM Therapy (Preclinical Stage)
Lead Therapy - Pompe Disease:
Our TRAM procedure uses a T-cells harvested from the patient (autologous), which are then modified with rapamycin and genetically engineered to systemically secrete the missing protein. Specifically in Pompe, we harvest T cells from the patients blood and treat them with rapamycin to induce a T-reg-like state (“T-rapa” cells). Then we engineer the T-rapa cells to express a working copy of the GAA gene which allows the cells to secrete the missing acid ceramidase. Our preclinical studies show significant capacity to restore function in cellular models (PMID 35298086). Preparations are underway to bring this therapy to the clinic.
Our platform technology can be applied to a variety of other diseases where therapeutic cross-correction can provide patient benefit. Contact us for more information.