![]() ![]() “In addition to improved efficiency of our RNA Gene Writers in PKU and SCD in preclinical models, we now have preclinical proof of concept data demonstrating their ability to correct the PiZ mutation responsible for alpha-1 antitrypsin deficiency, a serious genetic disease affecting the lungs and the liver with no definitive treatment. “We have made significant strides across our Gene Writing and non-viral delivery platforms,” said Michael Severino, M.D., CEO of Tessera Therapeutics. These new data are being highlighted in an invited symposium talk, three oral presentations and a poster presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles, California, May 16 – 20, 2023. Additional data were presented on multiplexed full-length gene writing and rewriting to generate tumor-killing CAR-T cells as well as robust delivery efficiency to HSCs and T cells. SOMERVILLE, Mass., (GLOBE NEWSWIRE) - Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today presented progress across multiple platforms and preclinical programs including increased efficiency in correcting the SCD mutation in preclinical models, increased efficiency in correcting the most common mutation in PKU in mouse and NHP models, and a first-time demonstration of efficient in vivo rewriting in AATD. Cecilia Cotta-Ramusino, Tessera’s Head of Platform, to highlight these data in an invited symposium presentation today at 8:50 a.m. ![]() Proprietary lipid nanoparticle (LNP) delivery demonstrates potential to effectively reach cell types beyond hepatocytes including hematopoietic stem cells (HSCs) and T cells.RNA Gene Writing™ technology enables multiplexed full-length gene writing and rewriting with RNA-only delivery to engineer CAR-T cells with robust antitumor activity.RNA Gene Writing™ platform demonstrates continued advances in rewriting to correct pathogenic mutations responsible for phenylketonuria (PKU), alpha-1 antitrypsin deficiency (AATD) and sickle cell disease (SCD) including proof of concept in non-human primates (NHPs) for PKU. ![]()
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