# Core C: Correlative Sciences PDCS/TCSL

> **NIH NIH P01** · UNIVERSITY OF PENNSYLVANIA · 2021 · $506,950

## Abstract

SUMMARY CORE C: CORRELATIVE SCIENCES 
Immuno-gene therapies have demonstrated an unparalleled anti-cancer efficacy in very poor prognosis 
patients. Limitations in the in vivo efficacy of the patient’s own T cells in CLL, antigen escape-related relapse in 
ALL, a CAR-engineered T cell pool outpaced by tumor cells in myeloma, and lack of appropriate, tumor- 
selective CAR in AML has given rise to the current proposal wherein innovative approaches are expected to 
solve the aforementioned challenges in cellular immunotherapy. Our trials will be the first to use 
CRISPR/Cas9-enhanced immune-gene therapy to treat some of the most challenging cases in each clinical 
indication; the first to use CART cells targeting two different antigens; the first to target AML with normal donor 
CART cells targeting CD33 while repopulating the host with CD33-edited hematopoietic stem cells. 
Critical to the success of cell therapies is a central correlative sciences laboratory to study the in vivo kinetics 
and homing of the infusion product(s) and the response of the tumor and the bioactivity of the infused cells; 
Core C provides this GCLP-based yet nimble infrastructure to support the clinical trials proposed herein, where 
SOP-specified molecular, cellular, and biochemical assays will be performed, reviewed by qualified personnel, 
and entered into a database for aggregate analysis to evaluate the efficacy of our proposed trials and compare 
with previously run CART cell trials at UPenn.

## Key facts

- **NIH application ID:** 10245070
- **Project number:** 5P01CA214278-05
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** SIMON F LACEY
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $506,950
- **Award type:** 5
- **Project period:** 2017-08-15 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10245070

## Citation

> US National Institutes of Health, RePORTER application 10245070, Core C: Correlative Sciences PDCS/TCSL (5P01CA214278-05). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10245070. Licensed CC0.

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