Long-acting Gene Therapy - One Dose Providing Years of Relief

Using a patient’s own cells modified to restore gene function

At Glafabra Therapeutics we use patient living cells to deliver our Live-cel Therapy to the patient. Patients using Live-cel therapies obtain freedom from the tedium of traditional ERT (enzyme replacement therapy). We harvest patient own cells and genetically-engineer them to express a missing enzyme. Reintroduction of the modified cells back into the patient achieves a therapy that can last at least 5 years after a single infusion (PMID 39794302). Glafabra is launching plans to apply their successful Live-cel Therapies approach to a wide variety of enzyme deficiency disorders.