Future efforts will focus on developing standard operating procedures (SOPs) for SV-AUC to enhance its reproducibility to match that of SEC-MALS.

However, the study did not assess the implications of these findings on current or future gene therapy research, nor did it provide any regulatory recommendations.

Engineered adeno-associated viruses (AAVs) are often utilized in gene therapies to deliver therapeutic genetic material directly to target cells, making accurate measurement of these vectors crucial for ensuring safety and efficacy.

Gene therapy is a rapidly evolving field that involves replacing or modifying faulty genes to treat diseases, with around two dozen products currently available and hundreds of clinical trials underway.

Researchers advised against using PCR-ELISA for quantitative measurements of AAV vectors without further development and standardization.

Additionally, A260/A280 dual wavelength UV spectrophotometry was found to have significant limitations, including its inability to distinguish between partially filled or overfilled AAV vectors, rendering it unreliable for precise measurements.

While SV-AUC is considered the 'gold standard' for measuring AAV vectors, the study found it to be less accurate than SEC-MALS, although it provided detailed mapping of genetic and viral particle distribution.

SEC-MALS emerged as the most accurate and precise measurement method suitable for general implementation in the field.

The measurement techniques evaluated included PCR-ELISA, SEC-MALS, SV-AUC, and A260/A280 dual wavelength UV spectrophotometry.

Overall, the study emphasized the importance of understanding the limitations and uncertainties of different measurement techniques in gene therapy, highlighting the need for further development.

A recent study, conducted in collaboration with the National Institute of Standards and Technology, NIIMBL, and USP, involved six industry labs from the U.S. and Europe measuring AAV vector samples using four different techniques.

The study revealed that the commonly used PCR-ELISA method is problematic, demonstrating the lowest accuracy and precision, which raises concerns about its reproducibility across laboratories.