The RhiaPharmaceutical AAV technology platform
Since our founding almost 20 years ago and with more than 40 years of pioneering AAV research, our vision has always extended beyond therapeutic development. Making genetic medicine highly accurate, safe, and effective is vital to improving patient lives, and the science for finding missing or mutated genes to correct genetic disorders has greatly evolved.
Equally important is ensuring that no matter where they live, every patient in need has cost-efficient access to genetic medicine. It is why we consider our AAV technology platform our greatest patient innovation. Its components are shaping how we scale gene therapy manufacturing and lower cost.
It is this uncompromised vision that propelled the emergence of a world-class gene therapy technology platform.
AAVPlatform proven end-to-end capability results in technology to increase scale and lower costs of goods as well as translate into clinic faster, safer and with greater predictability.
Three essential components of the RhiaPharmaceutical AAV platform
Capsids
A vast range of chimeric constructs to match any tropism and maximize transduction
Promoters
An expression cassette with gene control, regulation and inducible technology
Production
Pro10™, a high-yield suspension cell line with synthetic materials output, lowers cost
Ask Jude
“Gene therapy is changing lives. Overcoming the challenges of production, accessibility, and cost must also be a priority.”
R. Jude Samulski, PhD
Chief Scientific Officer and Co-Founder