Developing Nonviral Gene Therapy for Hemophilia A

“PVIII-101 is for hemophilia A, and it uses a nonviral transposon base system... An advantage of the nonviral delivery system is... you don't have the seroprevalence issue that traditional AAVs have. We have the potential to avoid many of the toxic and immunogenicity issues that we've seen with the traditional therapies. That’s a significant advantage in terms of patient population. Traditional AAVs in hemophilia today are all targeting adult patients... We showed data this morning that has the potential to provide a functional cure to not only adults but juvenile patients and potentially newborns as well.”

Poseida Therapeutics is developing a nonviral gene therapy for treating hemophilia A which has the potential to address current issues with adeno-associated virus (AAV) gene therapies such as immunogenicity, delivery capacity, and restricted patient populations. Data on the preclinical therapy, P-FVIII-101, were presented at the 64th American Society of Hematology (ASH) Annual Meeting, held December 10-12, 2022, in New Orleans, Louisiana, by Brian Truong, PhD, research scientist, Poseida Therapeutics.

CGTLive spoke with Brent Warner, president, gene therapy, Poseida Therapeutics, to learn more about the potential advantages of a nonviral gene therapy in the hemophilia A space. He discussed the data presented that showcased P-VIII-101's ability to normalize factor VIII expression.

Click here to read more coverage of the ASH 2022 meeting.

REFERENCE

Truong B, Nguyen G, Schwerk J, et al. Sustained factor VIII activity following single dose of non-viral integrating gene therapy. Presented at: 64th ASH Annual Meeting, December 10-12, 2022, New Orleans, Louisiana. Paper #400.