# CORE C Technology Implementation/Development and Correlative Sciences

> **NIH NIH P01** · UNIVERSITY OF PENNSYLVANIA · 2024 · $787,787

## Abstract

CORE C: Summary Abstract
Core C is comprised of two groups: the Human Genome Editing Laboratory (HGEL) led by Dr. Friederike Herbst
and the Translational and Correlative Studies Laboratory (TCSL) led by Dr. Joseph A. Fraietta. Core C works
closely with Cores A and B to actively support the proposed P01 grant renewal with the following three Aims:
In Specific Aim 1: Process Optimization, Development and Product Characterization, Core C will provide
knowhow and consulting support to Core B to manufacture CRISPR-edited CAR T cells in a shortened
manufacturing process (Project 1: CD5 knockout anti-CD19 CAR T cells). Core C will conduct scale-up studies
to optimize base editing of T cells and matching HSPC cell populations for further lockdown of manufacturing
processes to tech transfer to Core B (Project 2). Core C will develop a pipeline for subsequent characterization
of the gene edited cell products (T cells and HSPC) including assay developmental procedures important for
product safety and release testing, in line with FDA regulations for future IND applications. In Specific Aim 2:
Support of Clinical Trials Proposed by the 3 Projects, Core C will support all three clinical trials within this
grant proposal. Project 1: CD5 knockout anti-CD19 CAR T cells in patients with relapsed or refractory lymphoid
malignancies. Project 2: CD45-edited CART45 in tandem with CD45-edited matching HSPCs in patients with
acute myeloid leukemia; and Project 3: combination of MIL- and PBL-derived anti-BCMA CAR T cells in patients
with relapsed/refractory multiple myeloma. In addition to supporting the manufacturing Core B with product safety
analysis and product release assays testing for on-/off target editing, transduction efficiency, replication-
competent lentivirus and mycoplasma, Core C will perform a range of sample processing, biobanking and
correlative assays and data collection that will be tailored to the individual needs of each of the clinical trials. In
Specific Aim 3: Analytical Platform Development, Core C will strengthen three assay platforms identified as
being key correlative assay technologies needed to support the projects and clinical trials in this grant proposal.
Overall, all platforms - once established - will allow the identification as well as the molecular and phenotypic
characterization of rare subpopulations of therapeutic cells. Moreover, the generated data package will be
evaluated and utilized to predict and improve patient outcome. 1. Single cell transcriptome analysis to expand
correlative analysis by extending established protocols and existing data to optimize single-cell methods on
samples drawn from internally biobanked accessions. 2. Digital PCR using a new QIAcuity One plate-based
system to perform sensitive standard-independent quantification of rare sequences and support the trials with
assessment of gene editing and gene marking. 3. Flow cytometry Developing new 28+ color panels on a
spectral cytometer to be used for retros...

## Key facts

- **NIH application ID:** 10931433
- **Project number:** 5P01CA214278-07
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** Friederike Herbst-Nowrouzi
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $787,787
- **Award type:** 5
- **Project period:** 2017-08-15 → 2028-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10931433

## Citation

> US National Institutes of Health, RePORTER application 10931433, CORE C Technology Implementation/Development and Correlative Sciences (5P01CA214278-07). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10931433. Licensed CC0.

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