Awards, Research

QB3 director David Schaffer elected to National Academy of Inventors

Schaffer pioneered the use of directed evolution to precisely target viruses and the gene therapies they carry to specific tissues in the body

portrait of David Schaffer

David Schaffer, newly elected fellow of the National Academy of Inventors. (UC Berkeley photo by Mark Joseph Hanson)

David Schaffer, a UC Berkeley chemical engineer who pioneered the use of engineered viruses to deliver gene therapies — nine of his therapies are currently in clinical trials — has been named a fellow of the National Academy of Inventors (NAI), the highest professional distinction accorded solely to academic inventors.

Schaffer was one of 164 new fellows announced today (Dec. 7), all of them “academic inventors who have demonstrated a spirit of innovation in creating or facilitating outstanding inventions that have made a tangible impact on quality of life, economic development and the welfare of society,” according to an academy statement.

Schaffer is the Hubbard Howe Distinguished Professor at UC Berkeley and director of the Berkeley outpost of the California Institute for Quantitative Biosciences, known as QB3-Berkeley. He has appointments in the departments of chemical and biomolecular engineering, bioengineering, and molecular and cell biology, and he formerly was head of the campus’s Stem Cell Center.

“I’m thrilled to become a part of such an esteemed group of colleagues, including fellows from our campus and beyond,” Schaffer said. “This recognition motivates me to keep pushing to translate the technologies we’ve developed at Berkeley into therapies to treat human disease.”

The holder of 28 issued patents in gene therapy and stem cell therapy and co-founder of six startups, Schaffer is a pioneer in the development of novel gene and cell therapies to treat currently incurable human disease. Those currently in clinical trials include therapies for eye diseases that lead to low vision or blindness — wet age-related macular degeneration, diabetic macula edema, x-linked retinitis pigmentosa, choroideremia and late stage retinitis pigmentosa — and a rare disorder, Fabry disease, that also affects the heart, kidney and other tissues.

The virus that carries these therapies into patients — adeno-associated virus (AAV) — is benign in humans and easily invades human cells. Physicians have been using it for gene therapy for more than a decade, filling it with genes that the virus delivers into the cell to correct a genetic condition.

Schaffer’s innovation was to find a way to more precisely target the virus to a specific type of cell. For this, he used directed evolution: the repeated generation of genetic variants of AAV and selection of the most highly optimized and targeted vector for a particular tissue. As a result of his technique, AAV has emerged as the dominant delivery vehicle in the gene therapy field.

Schaffer founded the startup company 4D Molecular Therapeutics, now a public company, that has taken his patented technology into five human clinical trials. Schaffer cofounded four other companies focused on protein biologics, genome editing and stem cell therapies, and one, subsequently sold to Pfizer, that focused on oncolytic virus engineering.

In 2020, Schaffer was appointed director of QB3-Berkeley, a center focused on fostering innovation and entrepreneurship. In this role, he is developing programs to educate and mentor graduate students, postdoctoral fellows and faculty about the translation of technologies into companies to maximize societal benefit — a main focus of his career.

He has been recognized with numerous awards, including the Andreas Acrivos Professional Progress Award, the American Institute of Chemical Engineers Pharmaceuticals and Biotechnology Award, the American Chemical Society Marvin Johnson Award, the Biomedical Engineering Society Rita Schaffer Award, a National Science Foundation CAREER Award, and an Office of Naval Research Young Investigator Award. He also is a fellow the American Association for the Advancement of Science and the American Institute of Medical and Biomedical Engineers.

The new NAI Fellows will be inducted during a ceremony at NAI’s 11th annual meeting this June in Phoenix, Arizona.