22 Jul Tessera Therapeutics
Vikram Ranade, Ph.D., Senior Vice President, Corporate Development
Oct. 9 | 9:45am | FLW Ballroom G
Boston, Massachusetts
Tessera Therapeutics, Inc. is pioneering Gene Writing™—a novel genome editing technology that writes therapeutic messages into the genome to treat rare diseases and cancer. Our RNA Gene Writers can make permanent small and large alterations to the human genome using non-viral delivery of RNA molecules. We recently presented data at ASGCT that demonstrated our RNA Gene Writers are 1) highly efficient at in vivo correction of the mutation responsible for alpha-1 antitrypsin deficiency (AATD) to wild-type, 2) able to achieve therapeutically relevant levels of hemoglobin beta (HBB) rewriting in vivo in humanized mice when combined with our proprietary lipid nanoparticle (LNP) delivery – we believe these results can enable true correction to wild-type of the HBB locus responsible for sickle cell disease (SCD) and 3) can integrate CAR transgenes into T cells in vitro creating functional CAR-T cells that show tumor killing in vitro and in vivo.
