3D Tissue Imaging to Characterize Gene Therapies in Preclinical Drug Discovery

In recent years, gene therapies have shown exceptional promise in curing devastating rare genetic-based diseases by restoring normal cellular functions. With gene therapy approvals for diseases, such as childhood blindness (Spark Therapeutics, Luxturna) and spinal muscle atrophy (AveXis, Zolgensma), many novel candidates will be granted fast-track designation and eventual approval from regulatory agencies. For gene therapies to move from bench to bedside, visualizing the biodistribution of vector-mediated delivered genes is crucial in early-stage research studies.

In this webinar, we introduce cryofluorescence tomography (CFT), a unique 3D imaging modality that allows detection and visualization of vector-mediated gene expression across whole bodies and organs.

During the webinar, the speaker will:

• Provide an overview of the CFT technology and how it is used in gene therapy research
• Discuss how CFT addresses the challenges when looking to track the biodistribution of viral-based therapies
• Present multiple case studies to reveal how it is applied to evaluate various systems, such as AAVs and ASOs