Gene Therapy Analytical Methods
Few therapeutic modalities can match the excitement and promise of gene therapies. In recent years, they have generated life-changing results both in late-stage trials and increasingly, as FDA approved therapies. However, there remains a critical need in many more indications and the industry is facing mounting pressure to bring down the cost-per-dose.
One key component in optimizing gene therapy development is the analytical testing. Companies in this space need to find a partner that can meet the highest standards for safety and reliability, without slowing down commercialization timelines. Competition within rare disease communities is common and particularly critical in an era of one-time functional cures.
At Avomeen, we get it; diverse projects have diverse needs. Our flexibility and knowledge base allow us to customize our gene therapy analytical methods to suit each project’s needs. We help optimize and speed up processes, saving significant time and reducing costs as workflows begin to scale. Avomeen helps customers get solid data that clarifies the best development path forward. All the while, our QC mindset keeps us thinking ahead at each step, allowing for intelligent method development, to catch problems early and leapfrog challenges before they arrive.
|Strategy and commitment, ensuring a speedy gene-to-clinic strategy in an era of one-time functional cures.||Optimized method development, for lower manufacturing costs.||Robust data collection and documentation, to support one-of-a-kind regulatory paths.|
Gene Therapy Capabilities:
Manufacturing the doses required for a therapeutic effect is incredibly expensive, so efficiencies throughout gene therapy process development can significantly cut costs and increase the program’s viability. We work with our partners to determine what assays, methods, and instruments will best help them reach their goals. This may include:
- Viral vector purity determination
- Peptide mapping
- Vector genome and total viral particle titer determination by SDS-PAGE or capillary gel electrophoresis
- Potency and transduction method development
- Empty/full capsid determination by UPLC
- Residual polyethylenimine, iodixanol, benzonase, and host cell protein detection
- Capillary electrophoresis (CE, Protein Simple Maurice)
- Ultra-performance liquid chromatography (UPLC, Agilent) with ELSD, UV-DAD and FLD
- High-performance liquid chromatography (HPLC, Agilent) with UV-DAD, Sedex, Varian, ELSD
- Dionex ICS-3000 Ion Chromatography System
- Seivers Total Organic Carbon Analyzer
- Agilent 7500ce ICP-MS
- AB Sciex Triple Quadrupole API 2000 and 3000 Mass Spectrometers
- Waters Acquity UPLC with Xevo G2-S Quadrupole Time of Flight (QTOF)
- Mammalian cell culture facility
- Caron stability chambers (custom settings)
- Metrohm 899KF Karl Fisher Coulometer
- Perkin-Elmer Spectrum 65 and Spectrum 100 Fourier Transform Infrared Spectrometers (FTIR)
- Perkin Elmer Lambda 365 UV/Vis Spectrophotometer
- Fluorescent & UV plate readers
Safety in Gene Therapy Process Development
Undoubtedly, one of the biggest challenges to the clinical success of gene therapies remains patient safety (especially after the death of pioneering participant Jesse Gelsinger in 1999). The field has since recovered and more than 1,000 clinical studies are now underway, targeting a range of ailments, including cancer and monogenic diseases like 𝛽-thalassemia and sickle cell anemia. So, while an efficient in vivo or ex vivo vector-strategy is important for successful genetic transfer, these considerations come second to patient safety.
A careful analysis of viral vectors and transduction methods remains vital. Avomeen understands that accurate viral vector analysis (such as with AAVs) and development are essential to this process. We also understand that scientific confidence goes a long way towards ensuring gene therapies can hit their mark and cure difficult diseases.