Allergan/Editas finally kick off CRISPR trial — marking first in vivo test of the gene-editing technology
More three years after Allergan $AGN dipped its toes into the world of CRISPR/Cas9 gene editing by enlisting Editas $EDIT as a partner — the companies are now off to the races with a trial in patients with a rare eye disease.
This trial marks the first instance of using the technology to edit DNA within the body.
Meanwhile, partners Vertex $VRTX and CRISPR Therapeutics $CRSP kickstarted a trial earlier this year that employed the technology to engineer a gene-edited stem cell therapy to treat patients suffering from severe hemoglobinopathies, a group of blood disorders. Sangamo Therapeutics $SGMO has tested its older zinc finger technology in patients with two lysosomal storage diseases, but a setback diffused enthusiasm for the program — but the company bounced back with encouraging early data on its hemophilia program partnered with Pfizer.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.