Poseida presents preclinical data from P-FVIII-101 gene therapy for hemophilia A

Poseida Therapeutics (PSTX) announced that the company will present preclinical data from its P-FVIII-101 gene therapy program, partnered with Takeda (TAK), at the 2022 American Society of Hematology Annual Meeting being held in New Orleans and virtually December 10-13, 2022. The data establish preclinical proof of principle for the treatment of Hemophilia A using P-FVIII-101, a non-viral liver-directed gene therapy utilizing Poseida’s Super piggyBac delivery system, which could potentially lead to a functional cure. P-FVIII-101 utilizes the company’s non-viral, nanoparticle-based delivery system together with SPB, which enables increased transgene cargo capacity, stable integration into the genome, potential for re-dosing, and potentially simpler manufacturing processes. The data to be presented show that P-FVIII-101 achieved and sustained normalized hFVIII activity following a single dose and delivered therapeutic FVIII activity in mice following single and repeat doses, indicating the potential for dose titration. Durable responses were observed following a single dose reported over the study period of seven months. The data support that with SPB the therapeutic transgene expression cassette can be stably integrated into the genome of liver cells and provide consistent and durable therapeutic activity. In October 2021, Poseida announced that it had entered into a research collaboration and exclusive license agreement with Takeda to utilize the Company’s proprietary genetic engineering platform technologies for the research and development of gene therapies, including P-FVIII-101. The companies plan to continue preclinical studies to advance the program toward an Investigational New Drug application.