A Story of Perseverance: An Interview with Matthew Porteus

Physician-scientist Matthew Porteus, MD, PhD, has been a mainstay in the genome editing field for more than two decades. He trained at Stanford University Medical School before completing his residency and hematology/oncology fellowship at Boston Children’s Hospital/Dana Farber Cancer Institute. During his postdoctoral research at Caltech with Nobel laureate David Baltimore, Porteus began his journey in gene targeting, in particular homologous recombination, as a means to repair disease genes. He has been on the Stanford faculty since 2010, treating patients with sickle cell disease and other hematological disorders at the Lucille Packard Children’s Hospital. 

Porteus is a scientific co-founder of CRISPR Therapeutics, the company that launched the exa-cel CRISPR trial that culminated in the approval of Casgevy in December 2023. With the latest companies he has co-founded—first Graphite Bio, now Kamau Therapeutics— Porteus remains steadfast in translating the promise of homologous recombination to the benefit of patients with sickle cell disease. 

In this wide-ranging interview with GEN editorial director Kevin Davies, PhD, Porteus candidly discusses some of the scientific milestones along his 25-year genome editing journey, including an update on...