This level of efficiency was associated with an increase in serum concentration of the wild-type alpha-1 antitrypsin (AAT) protein and a significant decrease in aggregates of misfolded mutant AAT protein in mouse livers on histological examination.RNA Gene Writers achieved a 28% in vivo correction of the pathogenic PiZ mutation in a humanized mouse model of AATD.NHPs showed in vivo rewriting efficiencies at the PAH locus (the gene associated with PKU) of up to 45% in the liver, well above the 10% curative threshold.In a humanized mouse model of the R408W mutation (the most common disease-causing mutation for PKU), in vivo rewriting efficiencies of ~40% were observed at doses of 0.5 mg/kg, and in vivo rewriting efficiencies above the 10% expected curative threshold at dosages as low as 0.15 mg/kg.RNA Gene Writers Efficiently Make True Corrections of Causative Mutations in Multiple Models of Genetic Disease Tessera’s Head of Platforms, Cecilia Cotta-Ramusino, Ph.D., will discuss these data in an oral presentation titled “Writing DNA with RNA: Genome Engineering by Target Primed Reverse Transcription” today at 8:50 a.m. We look forward to capitalizing on this momentum to translate these novel genetic medicines into the clinic and to make the broadest possible impact for patients.” We are building a strong pipeline of Gene Writers designed to specifically address target indications and our continued progress is a testament to the power of our technology and the highly talented team we have here at Tessera. Michael Holmes, Ph.D., Chief Scientific Officer of Tessera added, “Our Gene Writing technology and delivery platforms continue to advance and demonstrate their broad potential to push the boundaries of what is possible in genetic medicine. Coupled with significant progress in engineering cancer-killing CAR-T cells using our multiplexing approach, we are excited to continue advancing our programs toward our goal of delivering cures to patients suffering from genetic diseases.” “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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