Gene Therapy

In another CRISPR first, Penn researchers dose US cancer patients with gene-edited T cell therapy

CRISPR has been tested in US patients for the first time — but perhaps not in the manner you imagined.

The University of Pennsylvania has led the way dosing two patients with relapsed cancers — one with multiple myeloma and one with sarcoma — with a T cell therapy in which the T cell receptor and PD-1 protein are edited out.

Usman “Oz” Azam

Funding for the trial came partly from Tmunity, a biotech co-founded by Penn’s CAR-T visionary Carl June and helmed by Novartis vet Usman “Oz” Azam. Parker Institute for Cancer Immunotherapy, an early investor in Tmunity, is also bankrolling the trial. A Penn spokesperson confirmed the news, which was first reported by NPR.

The basic idea here is not unlike CAR-T, except with different elements and technologies: Take a patient’s T cells, engineer it to express NY-ESO-1 TCR, while eliminating TCR and PD-1 originally on the surface using CRISPR.

A group of UCLA and UCSF scientists backed by the Parker Institute has been studying new techniques to get new genes into T cells — not just eliminating them — with CRISPR.

That’s not the most straightforward way of applying the gene editing tech, which has enticed other drug developers with the potential to elegantly fix genetic errors leading to disease. But it does speak to the versatility of CRISPR, now a popularized tool that’s spawned upstarts ranging from diagnostics to targeted bacteria eradication.

Carl June

Nevertheless, the trio of CRISPR pioneers associated — Editas Medicines, Intellia Therapeutics and CRISPR Therapeutics — remain the ones to watch for first-in-human testing. In fact, just two months ago CRISPR Therapeutics dosed the first patient with its Vertex-partnered beta thalassemia drug. It is also planning to treat a sickle cell disease patient with the same drug in mid-2019 for a separate trial.

Of course, that’s assuming we’re talking outside of China, where infusions of CRISPR-edited T cells in cancer patients have been taking place for more than a year. In a stunner, Chinese scientist Jiankui He claimed the birth of baby twins who were genetically modified as fetuses, stirring up widespread condemnation and soul searching in the scientific community.

“2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense,” Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley, told NPR.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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