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Caltech Together: Silicon Valley
June 19, 2025 at 11:30 am — 1:00 pm PDT
CRISPR and Gene Therapy: Join Us for a Luncheon Discussion
Date: Thursday, June 19
Time: 11:30 a.m.–1:00 p.m.
Location: Moore Foundation
1661 Page Mill Road, Palo Alto, CA 94304
To attend, please register here. Attendance is limited to 80.
If you would like lunch, please pay $30 by Monday, June 17, at 4:00 p.m. PT to our alumni volunteer David Adler via:
– Venmo (last 4 digits of his phone number: 7164)
– PayPal
We look forward to seeing you,
Peter Tong
CRISPR Revolution: The Next Chapter in Gene Therapy
With Matthew Porteus
Rewriting the genome to treat genetic diseases or develop new classes of drugs has been discussed for decades. Progress in the 2000s was limited—but the field transformed with the discovery of the CRISPR/Cas9 nuclease system, a bacterial mechanism used to fight infection.
The Nobel-winning discovery, published in 2012, was a test-tube experiment that simplified genome editing to two components: a protein that cuts DNA and a short RNA sequence that guides the protein to a specific genomic site.
Multiple investigators adapted this robust, highly specific, and easy-to-use DNA-cutting enzyme (nuclease) to treat patients with serious genetic diseases. In 2023, the first genome-editing drug, Casgevy, was approved.
Casgevy is now used worldwide to treat sickle cell disease and beta-thalassemia—two severe disorders affecting red blood cell function. Still, Casgevy represents just version 1.0 of genome editing.
I will discuss how we can harness CRISPR/Cas9 as a true word processor for the genome, not just a tool for correcting typos. I’ll also address current challenges in genome editing and gene therapy—and what the future may hold.
About the Speaker
Matthew Porteus, MD, PhD, is the Sutardja Chuk Professor of Definitive and Curative Medicine and a professor of pediatrics at Stanford University. He also holds appointments at Stanford’s Institute for Stem Cell Biology and Regenerative Medicine and the Maternal & Child Health Research Institute.
He serves as director of the Stanford Center for Definitive and Curative Medicine and co-executive director of the Laboratory for Cell and Gene Medicine, Stanford’s cell and gene therapy GMP facility.
Dr. Porteus was recently elected president of the American Society of Gene and Cell Therapy for the 2026–27 term.
His research focuses on developing genome editing as a curative approach to disease, especially hematologic conditions such as sickle cell disease, as well as diseases affecting other organ systems.
Dr. Porteus earned his undergraduate degree in history and science from Harvard University, where his honors thesis examined the recombinant DNA controversy of the 1970s. He completed his MD and PhD at Stanford, followed by clinical training in pediatric hematology/oncology at Boston Children’s Hospital and postdoctoral work with Nobel Laureate David Baltimore at Caltech.
In addition to his research, he continues to care for patients as an attending physician on the Pediatric Hematopoietic Stem Cell Transplant Service at Lucile Packard Children’s Hospital. He is committed to bridging clinical practice and research to develop innovative, curative therapies.
He has served on the 2017 National Academy Study Committee on Human Genome Editing and currently sits on the scientific advisory board for WADA on cell and gene doping. He is a scientific founder of CRISPR Therapeutics and a cofounder of Graphite Bio and Kamau Therapeutics, and sits on several other scientific advisory boards. Dr. Porteus is a strong advocate for developing transformative medicines in partnership with—and accessible to—global communities.
Our Alumni Volunteers
The following alumni work together to serve you: Avni Gandhi, Dave Adler, Jane Frommer, Mike Klein, Susan Huynh, and Peter Tong