Adeno Associated Viral Vector Characterization

Cryo-electron microscopy (cryo-EM) stands as the sole imaging method offering single-particle precision in pharmaceutical R&D for gene therapy. ATEM leverages proprietary software to enhance adeno-associated virus (AAV) characterization, a key vector in gene delivery.

ATEM’s cryo-EM technology boasts sample efficiency, requiring only 50 μl of sample at 10^12 particles, far less than the 500 μl at 10^13 particles needed by Analytical Ultracentrifugation, the existing gold standard.

This efficiency does not detract from the technology’s capacity to deliver detailed and validated analyses of AAV critical quality attributes (CQAs), distinguishing between full, empty, and intermediate AAV particles—a capability often lacking in traditional methods. This precision is crucial for gene delivery, as full particles contain the therapeutic genetic material.

ATEM’s software tools are in the process of gaining GMP certification, reinforcing their reliability and robustness. The cryo-EM technology also reveals other CQAs like particle aggregation and capsid integrity, enabling comprehensive, high-resolution characterization vital for ensuring batch consistency, refining production processes, and enhancing the safety and efficacy of gene therapies. Thus, ATEM’s cryo-EM, with its detailed, validated, and sample-efficient quantitative analysis, is poised to significantly influence gene therapy advancements in pharmaceutical R&D.