Assay Design for Ocular Biodistribution
A robust qPCR-based biodistribution study is built on a deep understanding of vector biology and tissue-specific challenges. The selection of an AAV serotype with the appropriate cellular tropism is a key variable influencing therapeutic outcomes. For instance, studies have shown how different pseudotyped AAV vectors exhibit distinct transduction efficiencies in specific tissues, underscoring the need for empirical biodistribution data to confirm vector performance in a given therapeutic context (PMID: 22937069).
Our approach incorporates:
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Optimized DNA Extraction: Protocols tailored for small, delicate ocular tissues to maximize DNA yield and purity.
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Validated Primer-Probe Sets: Assays targeting unique sequences within the vector cassette (e.g., promoter, transgene) to ensure specificity.
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Reference Gene Normalization: Use of validated, stable reference genes to calculate VGC per diploid genome.
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GxP Compliance: All assays are developed, qualified, and executed within a GxP framework to ensure data integrity for regulatory submissions.
Precise quantification of vector delivery supports safety assessment and confirms the viability of a therapeutic platform. The successful use of AAV vectors to deliver genetic payloads for reconstituting functional human cells in preclinical models demonstrates the importance of confirming vector delivery to validate a biological hypothesis (PMID: 24516613).
“We started collaborating with UPenn Vector core in 2023 and the AAV vector which they manufactured laid a foundation for development of a gene therapy candidate which will enter soon preclinical studies. The key people from UPenn Vector Core joined Franklin Biolabs and our partnership transitioned without interruption from UPenn Vecor Core to Franklin Biolabs Research Vector Division. Franklin Biolabs is our trusted partner in our AAV-vector based gene therapy candidate development and we hope to continue the partnership for years to come.”
— Biotech Partner
