On November 3, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that preclinical data from its P-FVIII-101 program, partnered with Takeda, has been selected for an oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, being held in New Orleans and virtually December 10–13, 2022 (Press release, Poseida Therapeutics, NOV 3, 2022, View Source;exposition-301667029.html [SID1234623055]).
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P-FVIII-101 is a liver-directed gene therapy product candidate for the in vivo treatment of Hemophilia A utilizing the Company’s non-viral piggyBac DNA Delivery System combined with its nanoparticle delivery technology. Compared to current generation gene therapy approaches that use traditional adeno-associated virus (AAV), the Company’s non-viral gene delivery strategy is designed to enable potential single treatment cures with lower toxicity to mitigate safety issues, allow for re-dosing if needed and deliver optimized FVIII transgenes without cargo size limitations.
The oral presentation will highlight preclinical data from initial proof-of-concept studies, which demonstrated that a single administration of P-FVIII-101 resulted in durable expression of the Factor VIII protein at therapeutic levels in a dose-responsive manner. Expression of Factor VIII was shown to be sustained over six months in mouse models. These data illustrate the potential of P-FVIII-101 utilizing piggyBac to provide a long-term durable response for the treatment of Hemophilia A, early in life, with stable integration.