The New Age of CRISPR

The federal biosafety and ethics panel has unanimously approved the first study in patients of the genome-editing technology CRISPR-Cas9, in an experiment that would use CRISPR to create genetically altered immune cells to attack three kinds of cancer.

It had been widely expected that the first human use of CRISPR would be a 2017 clinical trial by Editas Medicine, which announced last year that it plans to use CRISPR to try to treat a rare form of blindness called Leber congenital amaurosis. Only a few hundred people in the US have that disease. The possibility that a study siccing CRISPR on cancer will happen first suggests that the revolutionary genome-editing technology might be used against common diseases sooner than once thought.

The experiment, proposed by scientists at the University of Pennsylvania, still needs the approval of the medical centers where it would be conducted, as well as from the Food and Drug Administration, which oversees the use of experimental treatments in people. If the study gets those OKs, it would enroll patients with multiple myeloma, melanoma, and sarcoma, and be funded by the Parker Institute for Cancer Immunotherapy, which was launched this year by tech mogul Sean Parker.

The proposed early-stage clinical trial with 15 patients would gauge the safety of the experimental therapy and see how feasible it is to manufacture genetically engineered and CRISPR’d T cells. The scientists would remove T cells, which normally target cells that are “foreign,” like bacteria, from patients with multiple myeloma, melanoma, or sarcoma. They would then use CRISPR to genetically modify the T cells so that, infused back into a patient, they can target and destroy tumor cells.

The trial would be conducted at MD Anderson Cancer Center (enrolling nine patients) in Texas, and the University of California, San Francisco (three), as well as Penn (three). Penn would also produce the genetically modified T cells.

In a technique that several companies are competing to commercialize, traditional genetic engineering alters T cells extracted from patients so that the cells produce a “chimeric antigen receptor,” or CAR.

Once the T cells are infused back into patients, that CAR lets the cells find molecules called antigens, which protrude from tumor cells, like a key fitting a lock. If all goes well, the T cells would destroy the tumors. In particular, the T cells would glom onto the antigen NY-ESO-1.

For more information on CRISPR, join me at Cell & Gene Therapy Bioprocessing & Commercialization Expo this October in Boston. And for special savings, be sure to use the VIP code “B16188BLOG” – See you this October.




Justin Gaines is a Boston-based drug discovery professional and biotech enthusiast. During his tenures he has remained active within the biomedical engineering and regenerative medicine sectors, and has remained an avid proponent of bringing together the Boston life science community. He is currently leading the Bio Pharma Networking Group across the North East – a professional life science and healthcare community spanning 6K members.



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