Improving Gene Therapy Design and Manufacturability Using Synthetic Biology and AI

Broadcast Date: Tuesday, November 12, 2024
Time: 8:00 am PT, 11:00 am ET, 17:00 CET

For adeno-associated viral gene therapies to scale, scientists must first address safety and manufacturability challenges. Improving payload design and optimizing production systems can help scientists address those challenges and ensure the safety, efficacy, and scalability of their therapies.

In this GEN webinar, Mark Stockdale will discuss how the application of synthetic biology and artificial intelligence can be used to improve the safety, potency and manufacturability of gene therapies, and introduce AAV Edge, a comprehensive platform for end-to-end gene therapy optimization—from payload design to production cell lines and model-guided processes.

Key topics that will be covered in the webinar include:

• How an in-house transformer-based model is used to design tissue-specific promoters, resulting in a >200-fold dynamic range in expression between on-target vs. off-target tissues.
• How sequence optimization boosts expression for clinically relevant transgenes up to 7-fold.
• How silencing of transgene expression in the production cell line can reduce cellular stress and toxicity during the manufacturing process.
• How the combination of Asimov’s clonal HEK293 cell line, optimized two-plasmid system, and model-driven process development achieves unconcentrated titers of up to 1E12 viral genomes per milliliter (vg/mL) across multiple serotypes.

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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