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.
Allergan and Editas had originally expected to file an application to test their therapy in humans by the end of 2017, but manufacturing trouble pushed that timeline to October 2018. To get the FDA endorsement, Editas fought off a stiff patent challenge — and like its peers — batted back persistent questions about off-target effects to adequately convince the regulator it was armed and ready to make the jump from animal studies to humans.
The trial, christened Brilliance, will evaluate their experimental therapy AGN-151587 (EDIT-101) for the treatment of Leber congenital amaurosis 10 (LCA10), a rare, inherited form of blindness caused by mutations in the CEP290 gene.
LCA is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes, and are considered the most common cause of inherited childhood blindness, occurring in two to three per 100,000 live births globally. The most common form of the disease, LCA10, accounts for approximately 20% to 30% of all LCA patients.
Spark Therapeutics’ approved gene therapy Luxturna, which delivered via a viral vector, is tailored to treat another rare eye disease due to mutations in both copies of the RPE65 gene. The FDA nod sparked Roche’s $4.3 billion takeover of the company, although the deal is yet to be consummated.
The Brilliance study is set to enroll 18 patients — and include up to five cohorts and evaluate three doses of the drug. Each patient will receive a single dose of AGN-151587 administered via an eye injection aimed at photoreceptor cells in the retina.
Editas’ senior team has seen upheaval in the past year. In January, CEO Katrine Bosley, who led the company for nearly five years, announced her exit and severed ties with the board, without explanation. Chief financial officer Andrew Hack announced his decision to leave by March, back in December 2018, and chief medical officer Gerald Cox jumped ship at the end of last year.
Beset by setbacks, Allergan’s Brent Saunders made the best of a bad situation by agreeing to be swallowed by AbbVie $ABBV in an eye-watering $63 billion deal earlier this year. The US drugmaker was swayed by the potential of Allergan’s blockbuster Botox franchise to stabilize its revenue as its $20 billion cash cow Humira hurtles towards a patent cliff in the United States.
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