[ad_1]
CRISPR has been tested in American patients for the first time – but maybe not in the way you imagined.
The University of Pennsylvania opened the way by administering to two patients with recurrent cancers – one a multiple myeloma and the other a sarcoma – a T cell therapy in which the T cell receptor and PD-1 protein are eliminated.
Usman "Oz" Azam
The funding for the trial came in part from Tmunity, a biotech co-founded by Penn's visionary, CAR-T, Carl June, and led by veteran Usman "Oz" Azam of Novartis. The Parker Institute for Cancer Immunotherapy, one of the first investors in Tmunity, is also funding the trial. A spokesman for Penn confirmed the news, which was reported for the first time by NPR.
The basic idea here does not differ from that of CAR-T, with the exception of different elements and technologies: take the patient's T cells, develop it to express the NY-ESO-1 TCR, while eliminating the TCR and PD-1 on the surface with CRISPR.
A group of scientists from UCLA and UCSF, supported by the Parker Institute, is investigating new techniques for obtaining new genes in T cells – not just for eliminating them – with CRISPR.
This is not the easiest way to apply gene editing technology, which has appealed to other drug developers who can gracefully correct genetic errors that cause disease. But this shows the versatility of CRISPR, a tool now popularized that has generated technologies that range from diagnosis to targeted eradication of bacteria.
Carl June
Nevertheless, the trio pioneers of the CRISPR partner – Editas Medicines, Intellia Therapeutics and CRISPR Therapeutics – remains the one to watch for the first test in humans. In fact, just two months ago, CRISPR Therapeutics administered the first patient beta-thalassemia drug associated with Vertex. It is also planned to treat a patient with sickle cell disease with the same drug in mid-2019 for a separate trial.
Of course, this assumes that we are talking outside of China, where CRISPR-modified T cell infusions in cancer patients have been taking place for over a year. Jiankui He, a Chinese scientist, has announced the birth of twin babies genetically modified as fetuses, generating widespread condemnation and introspection in the scientific community.
"2019 is the year when the training wheels stand out and the world discovers what CRISPR can really do for the world in the most positive sense," said Fyodor Urnov, gene editing scientist. at the Altius Institute for Biomedical Sciences in Seattle. the University of California, Berkeley, told NPR.
[ad_2]
Source link