# Platform for Rapid Expression, Purification, and Analysis of Patient-Specific Gene Therapy Products

> **NIH FDA R01** · NORTH CAROLINA STATE UNIVERSITY RALEIGH · 2021 · $581,729

## Abstract

ABSTRCT
By 2050, adeno-associated viruses (AAVs) will be established as the workhorse for in vivo gene therapy,
impacting 60 - 80 million patients per year worldwide, and the success of this gene therapy will rely on the ability
to manufacture application-/patient-specific AAVs affordably and rapidly. However, while in vivo and ex vivo gene
therapy products are advancing rapidly, their biomanufacturing technology has not seen comparable progress.
At present, manufacturers achieve the mandated product purity upon strenuous optimization - at the expense of
productivity and product efficacy and run multiple production batches to meet the clinical demand -with significant
delays and cost to patients (up to $1.2M per individual). To address the challenges faced by current AAV
biomanufacturing, we propose to develop an integrated purification and sensing technologies to accelerate
process design and optimization and make small-batch AAV production affordable to specific patient cohorts.
The technology is based on the integration of chromatographic adsorbents for isolation of “full” AAVs in flow-
through mode coupled with label-free impedimetric biosensors for quantifying AAV titer and full:empty ratio.
These technologies will enable the application of advanced analytics for the prediction of AAV therapeutic
efficacy by correlating process parameters with biomolecular-level CQAs of AAVs. The integrated purification
and biosensor technology will be demonstrated in a pilot process for producing patient-specific doses of rAAV2-
retro and scAAV9-CB-CLN6, which have been shown to correct brain and behavioral pathologies in CLN6-
Batten disease, a rare lysosomal storage disease.

## Key facts

- **NIH application ID:** 10414307
- **Project number:** 1R01FD007481-01
- **Recipient organization:** NORTH CAROLINA STATE UNIVERSITY RALEIGH
- **Principal Investigator:** Michael Daniele
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2021
- **Award amount:** $581,729
- **Award type:** 1
- **Project period:** 2021-09-01 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10414307

## Citation

> US National Institutes of Health, RePORTER application 10414307, Platform for Rapid Expression, Purification, and Analysis of Patient-Specific Gene Therapy Products (1R01FD007481-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10414307. Licensed CC0.

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