Pipeline Overview

The programs at Glafabra Therapeutics target a variety of metabolic disorders with our novel, long-lasting Live-cel 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 Live-cel Therapy (Clinical Stage)

Our STEM Live-cel therapy approach is an improvement over intermittent enzyme exposure with recombinant protein enzyme replacement therapy (ERT) . Unlike traditional ERT, our autologous-derived STEM Live-cel therapy provides uninterrupted cross-correction therapy - the patient obtains a more continuous supply of enzyme. The durability of our STEM Live-cell 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 Live-cel Therapy (Preclinical Stage)

Our TRAM Live-cel therapies 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 with TRAM Live-cel therapies show significant capacity to restore function in cellular models (PMID 35298086). Preparations are underway to bring this TRAM Live-cel 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.