# Nonhuman Primate Core

> **NIH NIH U19** · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2022 · $906,156

## Abstract

Core C: Project Summary/Abstract
Transplantation of hematopoietic stem and progenitor cells (HSPCs) underlies the only known case of HIV-1
functional cure. Over 12 years after this pioneering clinical case study, however, substantial improvements are
needed in order to apply this case study to a broader spectrum of HIV+ individuals. The overarching goal of our
U19 consortium is to identify candidate strategies to safely and effectively modify a patient's own HSPCs to resist
HIV infection, and simultaneously enhance their ability to recognize and destroy infected cells. The Nonhuman
Primate (NHP) Core will play a central role in organizing preclinical aspects of the approaches proposed by each
project in our group, maintaining a focus on translationally relevant features that are best suited for future trials
in patients. In Project 1 (Kitchen), we will generate a series of 12 NHPs that are transplanted with autologous,
gene-modified HSPCs. These cells will express a promising virus-specific immune effector molecule known as
CD4CAR, in combination with other promising therapies such as broadly neutralizing antibodies (bNAbs), and
therapeutic vaccination. CD4CAR-modified HSPC progeny, in combination with bNAbs and vaccine-specific
cells, represent a formidable and synergistic approach to target virus persistence. In Project 2 (Morizono), we
will focus on approaches to gene-modify HSPCs without removing them from the body, studying a total of 9
NHP. So-called “in vivo delivery” approaches will significantly enhance the applicability of anti-HIV gene
therapies to patients around the world. In Project 3 (An), we will test another important aspect in a total of 6 NHP:
the ability to precisely regulate the levels of gene-modified HSPCs and their progeny ex vivo and in vivo. Each
of the NHP Core's project-specific functions will be assessed with an eye towards the clinic, in close consultation
with our longstanding partners in Project 4 (Symonds). The goals set forth by the NHP Core are to implement
each of the NHP studies described above, produce gene-modified NHP HSPC products using potent lentiviral
vectors, and to provide supportive care for animals in each project, both following HSPC gene therapy and
infection with HIV-like viruses. Each of the four projects in our consortium are highly complementary. We will
bridge large animal studies overseen by each project, contribute meaningfully to discussions regarding synergies
between projects, and evaluate new and promising therapies as they emerge.

## Key facts

- **NIH application ID:** 10468650
- **Project number:** 5U19AI149504-03
- **Recipient organization:** UNIVERSITY OF CALIFORNIA LOS ANGELES
- **Principal Investigator:** HANS-PETER KIEM
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $906,156
- **Award type:** 5
- **Project period:** 2020-05-07 → 2025-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10468650

## Citation

> US National Institutes of Health, RePORTER application 10468650, Nonhuman Primate Core (5U19AI149504-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10468650. Licensed CC0.

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