About sAAVient Therapeutics

sAAVient Therapeutics is a clinical stage gene therapy company, focused on gene therapy of human liver diseases in general, and hemophilia B and hemophilia A in particular, using proprietary next generation (NextGen), generation X (GenX), and optimized (Opt) adeno-associated virus serotype 3 (AAV3) vectors. The remarkable specificity and efficacy of AAV3 vectors for human liver was first documented by aaVective scientists nearly a decade ago. Subsequently, aaVective’s NextGen, GenX, and Opt AAV3 vectors were shown to be highly efficient in human cells in vitro, and in mouse models in vivo. sAAVient’s NextGen AAV3 vectors have also been evaluated for their safety and efficacy in “humanized” mice and in non-human primates.

The first generation AAV5 and AAV8 vectors, composed of naturally occurring capsids, the two other serotypes currently being used by other AAV hemophilia gene therapy companies, have been shown to induce immune responses, especially at high doses since the host immune system cannot distinguish between AAV as a virus versus AAV as a vector, and since the wild-type AAV did not evolve for the purposes of delivery of therapeutic genes.

sAAVient Therapeutics utilizes the natural tropism of AAV3 for human liver, and in pre-clinical studies, NextGen AAV3 vectors have been shown to perform significantly better than first generation AAV5 and AAV8 vectors. Thus, in contrast to the high vector doses that are currently being used with other serotypes, the optimized AAV3 serotype vectors are likely to be more efficacious at lower doses. The Opt AAV3 vectors also offer the potential advantages of being less immunogenic, and more cost-effective, for their use in human liver diseases in general, and hemophilia in particular.

With the combined extensive experience of the scientific founders in AAV biology and vector development spanning more than four decades, sAAVient Therapeutics is ideally positioned to overcome the technological challenges that a number of other hemophilia gene therapy companies using less efficient, first generation of AAV serotype vectors have encountered and continue to encounter. The sAAVient Therapeutics team possesses novel insights into the basic biology of AAV vectors in general, and AAV3 vectors in particular, that promise to lead to a permanent cure of hemophilia with a one time treatment.