The Science of Hope: Exploring Cell Therapy

Speakers & Sessions

Carl June, MD: Director, CCI

Welcome
Brief introduction to the series and welcome remarks. (Welcome talk is linked within the Immune System session).

Daniel Goodman, PhD: Assistant Professor, Cancer Biology

Session: The Immune System
"Beyond protecting you from infections, your immune system has an important regulatory role in your body and plays a central role in many diseases, including cancer. This session introduces the key cell types and pathways of the immune system, and how innate and adaptive responses work together."

Andrew Rech, MD, PhD: Instructor, Pathology & Laboratory Medicine

Session: Cancer

Microscopic Battlegrounds: A Pathologist’s View of the Cancer-Immune War

Cancer isn't just a disease of rogue cells; it’s an active battlefield where the body's immune system constantly fights back. Join Andrew Rech, an Instructor and immunotherapy researcher from the University of Pennsylvania, for a visual journey into this microscopic war. We will explore the "Rules of Engagement"—how the immune system identifies and attacks tumors, and the clever ways cancer evades detection. We will look at cells in patient samples to visualize how pathologists determine if the immune system is winning, providing a foundational look at the science powering cutting-edge treatments like CAR T-cell therapy.

Julie Jadlowsky, PhD: Director, Translational Research Operations

Session: Cell Engineering - Part 1 (Sessions 1 and 2 are linked together here)
An introduction to types of cell therapies, therapeutic target selection, vector options for cell engineering, and an overview of validating safety for human administration

Andrew Fesnak, MD: Deputy Director, CACT

Session: Cell Engineering - Part 2
Good-manufacturing practices, manufacturing cell products for clinical use, quality control of engineered cell products

Elizabeth Hexner, MD: Medical Director

Session: Cell Therapy for Blood Cancers
This will be an interactive session introducing stem cell transplantation as the first cell therapy and the first immunotherapy. Using a patient’s journey with acute leukemia, we will introduce decision making about proceeding with a transplant, various sources of blood forming stem cells, and some of the complications that can be encountered.

Daniel Baker, PhD

Session: Cell Therapy for Solid Tumors
Dr. Baker will provide a comprehensive overview of state-of-the-art cell therapy strategies for solid tumors, including CAR T cells and other adoptive immunotherapies. He will delineate the principal obstacles posed by the tumor microenvironment and showcase emerging translational approaches devised to overcome these barriers.

Friederike Herbst-Nowrouzi, PhD: Director, HGEL

Session: Gene Therapy
In this session, students will explore how gene therapy treats genetic diseases by correcting or replacing defective genes.
We'll focus on sickle cell disease (SCD), a blood disorder caused by a single mutation, and learn how scientists are developing ways to "fix" this mutation using cutting-edge tools like CRISPR.
Students will also learn about lipid nanoparticles (LNPs), tiny bubbles that deliver gene-editing tools into cells. We'll highlight research from Dr. Musunuru’s lab, where LNPs were used to deliver a customized base editing machinery to an infant’s liver to correct a CPS1 mutation. This was the first personalized, in vivo CRISPR therapy in a human, and the patient, KJ, is doing remarkably well.
By the end of the session, students will understand the basics of gene therapy, why it holds so much promise for diseases like SCD or CPS1 deficiency, and how innovation in delivery technologies is shaping the future of medicine.

James Riley, PhD: Professor, Microbiology

Session: Cell and Gene Therapy for Indications Beyond Cancer

Success of CAR T cell therapy in cancer has sparked interest in how this approach can be used to treat other chronic conditions like HIV and autommune disease. This lecture will show how the principles of cancer CAR T cell therapy are being translated to other disease states.