June 21, 2016
By Alex Keown, BioSpace.com Breaking News Staff
SAN FRANCISCO – Silicon Valley entrepreneur Sean Parker’s new Cancer Institute is taking aim at cancer through the use of gene editing technology of CRISPR-Cas9. How that plays out will be determined by a safety review of federal regulators later today.
Parker Institute for Cancer Immunotherapy, founded in April with a $250 million endowment by the former tech giant of Facebook and Napster fame, is backing the planned testing of the gene editing technology on humans. The proposed testing will be under review today by a safety panel at the U.S. Food and Drug Administration and University of Pennsylvania researchers. If approved, the gene editing technology will be used to alter a patient’s blood cells, particularly the T cells, in order to kill the cancer cells. T cells are the soldiers of the body’s immune system. The proposed use of this particular trial would be to combat myeloma, melanoma, and sarcomas, MIT Technology Review reported this morning, citing a report by the National Institute of Health.
In addition to improving the body’s T cells through the CRISPR process, the technology will also be used to “edit out” the body’s PD-1 protein, a checkpoint of the immune system, MIT Technology Review said.
“In this first of its kind study, a team of Parker Institute investigators will utilize CRISPR gene editing technology with the goal of unleashing the power of the T-cell while simultaneously reducing the chance for autoimmunity. We’re excited to be part of the first clinical effort in the United States to combine these two powerful therapeutic approaches to treat a devastating disease like cancer, Parker Institute spokesperson Jennifer Haslip said in an email to Xconomy.
If the CRISPR project is given the greenlight, it would likely be a small trial, up to 15 patients, according to news reports.
At this time it is unknown how much of the Institute’s $250 million endowment will go toward the CRISPR project.
“CRISPR” refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the “molecular scissors” that cut and edit, or correct, disease-associated DNA in a cell. The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier. Doudna went on to found Caribou Biosciences. Charpentier sold her part of the rights of the CRISPR-Cas9 platform to CRISPR Therapeutics, a company making big moves in the gene therapy field.
CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions. Other companies that are directly controlling the technology include Caribou Biosciences, which has formed a strategic alliance with DuPont , and Novartis . Caribou also formed Intellia Therapeutics, which is active in gene therapy. In 2015, Juno Therapeutics struck a $727 million deal with Editas Medicine, the gene editing company, for a partnership that will forge three research programs marrying Editas’ technologies, including CRISPR-Cas9, to Juno’s CAR-T and TCR technologies.
Gene editing technology is making strides outside the United States. French biotech firm Cellectis and its development partner Servier, announced this week that it had begun dosing patients with a gene editing therapy known as TALENS, XConomy said. This is the first time TALENS has been part of a clinical trial.
Parker, who has a net worth of more than $4 billion, founded the Institute to focus on immunotherapy responses to cancer by bringing more than 40 cancer laboratories and more than 300 researchers to tackle cancer. The new model will provide additional funding for researchers while they continue to work at their home base of operations. In April, Parker told the Washington Post the model he envisions will allow researchers to collaborate without worrying about bureaucratic red tape.
