Lentiviral Gene Delivery

Lentiviral vector-based gene delivery calls for blood-producing cells to be harvested from the patient, transduced ex vivo with a healthy globin gene, and returned, corrected, to the patient to enable the re-constitution of bone marrow and a full cure.  Currently, bone marrow transplant is an effective long-term treatment for genetic blood disorders, but there is no match for 80% of patients, who will become the target market for LentiGlobin™ therapy.  GENETIX’ proprietary LentiGlobin™ vector is based on ten years of research at Harvard and MIT.  The vector carries the bA-T87Q-Globin gene, with the locus control region providing high, regulated expression levels. Safety elements (self-inactivation and chromatin insulators) prevent creation of replication-competent virus and unwanted gene activation.  In mouse models, RBC’s from transplanted human stem cells produce, on average, 35 ± 5% of normal human b-globin (up to 59%).