There are several recombinant viral vectors available for the delivery of therapeutic genetic cargo to the body’s cells. Recombinant adeno-associated virus vectors (AAV) have become the dominant gene-delivery vector of choice, aimed at curing or treating disease by delivering new or replacement genes to code for therapeutic proteins in humans. AAV offers the advantages of no pathogenicity, efficient long-term gene expression, ease of genetic manipulation, and the property of low or, in many cases, absent immune response. The Actus Therapeutics team includes pioneers in the development of AVV vectors, and our proprietary AVV vectors are a key differentiator for the company.


In partnership with AskBio, Actus has access to a proprietary manufacturing system that is both flexible and scalable. AskBio’s ability to support plasmid-based transfection of a patent-protected master cell bank provides cost and speed advantages at the early research stages of development, while also demonstrating performance that is scalable enough to support ongoing long-term clinical development needs. The manufacturing system is a production platform that overcomes flexibility barriers, allowing early feasibility benchmark work to be translated to clinical product development without necessarily replacing reagents or basic manufacturing methods.

AAV2/8-LSPhGAA (Pompe Disease Candidate)

Actus Therapeutics’ lead candidate for Pompe disease, AAV2/8-LSPhGAA, is a one-time gene therapy treatment that is designed be used with enzyme replacement therapy (ERT) to reduce immunogenicity. It also has the potential to correct GAA deficiency without the need for ERT. In 2017, Actus filed an IND for AAV2/8-LSPhGAA with funding from the National Institutes of Health. The trial will begin in 2018.

Market Opportunity

Pompe disease is a rare disease affecting approximately one in 40,000 people worldwide. There are currently no cures for the disease, but enzyme replacement therapy (ERT) has recently been introduced as the most effective option. Current ERT therapies have high rate of adverse events, as well as risk of severe allergic reactions, immune-mediated reactions and cardiorespiratory failure. Other limitations include a short half-life in the bloodstream, poor uptake and the requirement for repeated dosing (every two weeks). Currently, ERT costs up to $500,000 per patient per year, an estimated market of $700M per year. The current market for Pompe disease is predicted to grow at a rate of 11.5% between 2016 and 2020.