Allergan has taken up the option of developing and marketing a CRISPR candidate from Editas to treat a retinal degenerative disorder.
Building on its existing licensing options deal with the genome editing company Allergan has now confirmed it will co-develop and co-commercialise EDIT-101, which is part of the terms of the $90m deal.
That existing deal gives Allergan exclusive access, along with the option to license up to five of Editas’ genome editing programmes for ocular disease, including EDIT-101.
In conjunction with its option to co-develop and co-commercialise, Editas received $15m for EDIT-101, and is eligible for an additional $25m upon acceptance of an investigational new drug application by the FDA.
If all goes well, the companies are hoping that EDIT-101 will soon treat patients suffering with an eye condition called Leber Congenital Amaurosis 10 (LCA10), a disorder caused by mutations to a single gene.
It is the most common form of LCA, which is the most common cause of inherited childhood blindness, occurring in every 2-3 per 100,000 live births worldwide.
CRISPR-based medicines have the potential to be game-changers for patients with both genetically-defined and genetically-treatable diseases of the eye,” said David Nicholson, Ph.D., chief research and development officer, Allergan. “The Allergan team is excited to work with colleagues at Editas Medicine to develop EDIT-101 and potentially deliver a transformative medicine for LCA10 patients.”
“Today marks a significant milestone in our collaboration with Allergan and in our work to develop genomic medicines to treat eye diseases,” said Katrine Bosley, president and chief executive officer, Editas Medicine. “Allergan is a long-time innovator in ophthalmology, and their deep experience in developing, manufacturing, and commercialising medicines globally will meaningfully advance the EDIT-101 programme and maximise our ability to bring this transformative medicine to people living with LCA10.”
CRISPR, is a technology that can edit DNA at precise locations in the human genome, allowing researchers to permanently modify genes.
The technology has attracted huge interest and investment over the last few years, but recent news has knocked confidence in the approach.
Back in March, the US regulator halted Vertex Pharma’s planned trial involving CRISPR for sickle cell disease, which will likely delay the use of CRISPR-based therapies in humans.
Meanwhile a paper published in the journal Nature Biotechnology found the technique can cause unexpected genetic damage which could lead to dangerous changes in some cells.
This added to separate research which suggested the CRISPR gene editing might inadvertently increase cancer risk in some cells. These developments have caused the shares of leaders in the field, CRISPR Therapeutics, Intellia Therapeutics and Editas, to drop sharply.
However, Editas CEO Katrine Bosley remains optimistic, and her company’s partnership with Allergan will be one of the first tests of the long-term viability of the technology.