In Vivo T-Cell 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

Advances Towards In Vivo T-Cell Therapies (GlobeNewswire) - "Gene Writers formulated in LNPs can integrate CAR transgenes targeting CD20 in resting NHP T cells, achieving an average of 60% CAR writing in vitro; LNP delivery of Gene Writer and CAR template RNAs targeting B-cell maturation antigen (BCMA) to activated or resting T cells in vitro generated functional CAR-T cells that efficiently killed tumor cells in vitro and in vivo in tumor-bearing xenograft mice; Proof-of-concept studies in oncology demonstrated that with a single intravenous infusion of Gene Writer formulated in a proprietary LNP to tumor-bearing xenograft mice generated functional CD19-targeted CAR-T cells in vivo that were able to expand and eradicate tumor burden; LNP delivery of Gene Writer and CAR template RNAs targeting CD20 resulted in an average of 30% CAR writing in vivo in a naïve humanized mouse model containing resting T cells, which led to the elimination of human B cells in the blood compared to control..."

Preclinical • Oncology