# Core C: Viral vector core

> **NIH NIH U19** · SCRIPPS FLORIDA · 2020 · $314,375

## Abstract

PROJECT SUMMARY (Core C – Viral Vector Core)
The potential of recombinant adeno-associated virus vector (rAAV)-mediated delivery of HIV inhibitors with
broadly neutralizing activities to HIV infection is clearly demonstrated in work from Phil Johnson, Reed
Clark, and Ronald Desrosiers (Nature Medicine 2009), Alex Balazs and David Baltimore (Nature 2012),
Matthew Gardner and Michael Farzan (Nature 2015). The therapeutic potential of AAV-expressed inhibitors
is clearly shown in this proposal and recent published work from the Desrosiers Lab (Immunity 2019). The
main objective of the Vector Core is to provide high quality single-stranded (ss) and self-complementary (sc)
rAAV vectors to support the proposed studies. We will accomplish this goal through two specific aims. Aim
1 will design, create, produce, and quality control test scAAV vector lots at different scales with a variety of
capsids, transgenes and expression cassettes to serve the specific needs of other investigators of this
program project. 154 rhesus macaques will be enrolled for different studies over 4 years, and hundreds of
mice will be used for pre-macaque evaluation. On average, we estimate that -30 vector lots will be
produced annually to meet the needs of those studies. Aim will develop a novel and scalable rAAV
production method for larger scale translational NHP studies and future clinical development of rAAV-based
anti-HIV vaccine and therapeutics. Our current AAV production system should meet the vector needs in the
early stage of this program project. However, large scale vector production may become a bottle neck for
larger translational NHP studies and future clinical development. We will utilize our extensive experience in
developing various vector packaging cell lines, suspension cell culture-based vector production,
corresponding downstream processing and chromatography-based purification systems to develop a
scalable suspension 293 cell-based production method to overcome this limitation. In doing so, we further
our common goal of establishing AAV-mediated functional cures in macaques and humans.

## Key facts

- **NIH application ID:** 9891593
- **Project number:** 1U19AI149646-01
- **Recipient organization:** SCRIPPS FLORIDA
- **Principal Investigator:** Guangping Gao
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $314,375
- **Award type:** 1
- **Project period:** — → —

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9891593

## Citation

> US National Institutes of Health, RePORTER application 9891593, Core C: Viral vector core (1U19AI149646-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9891593. Licensed CC0.

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