The need for rare and ultra-rare genetic disease projects stems from the fact that over 95% of these conditions lack approved treatments, despite affecting hundreds of millions globally. Traditional drug development models are not feasible for ultra-rare populations, making it critical to establish baseline processes for small, geographically dispersed patient groups. Building standardized approaches for small cohorts not only accelerates therapy development but also creates a replicable model for future rare disease programs.
This project delivers an open-access, scalable framework for AAV vector manufacturing tailored to the unique challenges of rare and ultra-rare genetic diseases. By establishing a standardized baseline process, its our first stage at enabling efficient, reproducible production for small and geographically dispersed patient populations, where traditional manufacturing models are impractical. The approach creates a repeatable, adaptable model that can be applied across multiple rare disease programs, reducing development barriers, accelerating innovation, and ultimately expanding access to life-changing therapies.
Established a baseline for upstream and downstream processes, reducing variability and ensuring reproducibility for future GMP runs
Align analytical methods for critical quality attributes, enabling consistent product characterization and compliance readiness.
Implementing a pilot-scale, non-GMP viral vector production run provides manufacturers with a critical baseline for process development and analytical methods. This approach reduces future scale-up risks, accelerates readiness for GMP manufacturing, and optimizes cost-efficiency by adopting scalable purification strategies—potentially lowering labor and facility costs while minimizing contamination risks.
AAV bulk drug substance for future studies
Future: White Paper w/ process description Certificate of Testing ( CoT )*
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