Frequently Asked Questions (FAQ)

Pre-existing neutralizing antibodies (NAb) against AAVrh74 represent a significant exclusion criterion for Duchenne muscular dystrophy (DMD) clinical trials. To de-risk patient enrollment and ensure therapeutic efficacy, we have developed and validated a high-throughput, GxP-compliant NAb screening platform. This cell-based functional assay provides definitive go/no-go data for large patient cohorts, directly supporting accelerated clinical timelines.

The Enrollment Challenge: Pre-existing Neutralizing Antibodies to AAVrh74

For systemic AAV gene therapies targeting DMD, the presence of pre-existing NAb can abrogate vector efficacy and lead to patient exclusion. The AAVrh74 serotype, while promising, is subject to this challenge due to natural environmental exposure to related AAVs.

Identifying seronegative participants requires a screening platform that is not only sensitive and specific but also scalable enough to manage hundreds or thousands of patient samples without creating a bottleneck in trial recruitment. The logistical and regulatory demands necessitate a robust, validated bioanalytical solution.

A Validated, Scalable Platform for NAb Assessment

Our NAb assessment platform is built on a cell-based assay that quantifies the functional neutralization of AAVrh74 transduction. This approach provides a direct measure of inhibitory activity, which is the most clinically relevant metric for predicting in vivo response.

The operational expertise behind our vector analytics is a direct continuation of the foundational work performed by our scientific leadership. This continuity ensures a deep, specialized knowledge base for our partners.

“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

Our bioanalytical laboratories are part of a >100,000 sq ft facility designed for complex biologic development programs.

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Scientific Rationale and Assay Design

The accuracy of bioanalytical data is foundational to clinical success. The scientific principle of using high-fidelity tools to ensure precise biological characterization, as demonstrated in the context of isolating novel AAV genomes [PMID: 34448594], directly informs our assay validation philosophy. We apply this same rigor to ensure our NAb assays generate unambiguous data.

This level of precision is necessary to translate promising preclinical outcomes, such as the long-term dystrophin expression observed in large animal models of DMD [PMID: 22146342], into predictable human results.