Our mission is to transform gene therapy for human liver diseases

sAAVient Therapeutics was established in 2023. Our founders have more than 75 cumulative years of experience and hold 11 patents for innovations in the area of AAV3 vector development for liver-directed gene therapy.

AAV3 is the most efficient vector with a remarkably selective human liver-tropism – both for primary human hepatocytes and for primary human liver sinusoidal endothelial cells. sAAVient Therapeutics has developed capsid-modified NextGen AAV3 vectors that are up to 15-fold more efficient in transducing human liver cells. We've also developed genome-modified GenY vectors that are up to 10-fold more efficient in mediating transgene expression. sAAVient Therapeutics has documented the safety and efficacy of a clinical candidate NextGen AAV3 vector expressing human clotting factor IX (hFIX) in “humanized” mice and in non-human primates. NextGen AAV3-hFIX vectors will soon be evaluated in a Phase I/II clinical trial for severe hemophilia B.