In Vivo Sickle Cell Disease Therapy / Tessera Therapeutics - LARVOL DELTA

Tessera Therapeutics Features New Preclinical Data Demonstrating Progress Across its In Vivo Gene Writing™ Programs and Delivery Platform at the American Society of Gene and Cell Therapy 28th Annual Meeting (GlobeNewswire) - "Presented new preclinical data in non-human primates (NHP) for alpha-1 antitrypsin deficiency (AATD) and phenylketonuria (PKU), where RNA Gene Writer achieved an estimated 76% and 70% editing in hepatocytes1, respectively; New AATD data reinforced the safety and precision of Tessera’s proprietary LNP delivery system, demonstrating that it was well tolerated in NHP with high liver specificity and no off-target activity detected; Preclinical data for sickle cell disease (SCD) across mouse and NHP models highlight Tessera’s RNA Gene Writers’ ability to drive durable and efficient gene editing greater than 20% in long-term hematopoietic stem cells (LT-HSCs), reaching a potentially curative threshold"

Preclinical • Alpha-1 Antitrypsin Deficiency • Phenylketonuria • Sickle Cell Disease