The CRISPR patent interference case was newsworthy not because of the dispute, but because of the potential of the underlying technology.
As that uncertainty begins to clear, it’s time to get back to the scientific promise.
On Tuesday, Allergan Pharmaceuticals and Editas Medicine announced a new “strategic research and development alliance,” which grants Allergan exclusive access and an option to license up to five investigational programs for specific eye disorders.
Editas will receive an upfront payment of $90 million, with the potential to earn more if certain milestones are reached.
At the heart of the deal is Editas’ lead program for Leber congenital amaurosis (LCA), a family of rare, inherited retinal dystrophies. LCA is the most common cause of inherited childhood blindness, with a global incidence of two to three per 100,000 live births. Symptoms typically begin within the first year of life, leading to substantial vision loss and blindness.
Across the 13 subtypes identified, scientists have isolated disease-causing mutations in at least 14 different genes. Editas has focused its efforts on LCA10, a subtype driven by a defect in the CEP290 gene that accounts for some 20-30 percent of cases.
As a gene-editing technique, CRISPR could theoretically be used to correct the faulty gene early in life, lessening or preventing the dystrophy (wasting) of the retina and the resulting loss of vision. If the fundamental mutation is corrected, there’s a hope that the response could last for the rest of the patient’s life.
Whether or not that will eventuate remains to be seen. Based in Dublin, Ireland, Allergan has a well-established portfolio of ocular therapies. But all five Editas programs are preclinical and the CRISPR field only goes back four or five years.
Editas seems confident that the new partnership will help it achieve long-term success.
“Working together with Allergan through their Open Science R&D model significantly enhances our ability to develop genome editing medicines to help patients with serious eye diseases,” said Katrine Bosley, president and CEO of Editas in a joint news release. “This alliance is highly aligned with our strategy to build our company for the long-term and to realize the broad potential of our genome editing platform to treat serious diseases.”
Several weeks ago, Boston, Massachusetts-based Exonics Therapeutics launched with $5 million in seed funding from CureDuchenne Ventures, the investment arm of a non-profit Duchenne muscular dystrophy (DMD) advocacy group. While the research is early-phase, the ultimate aim is to deliver a single treatment to DMD patients, correcting the faulty gene that drives their debilitating disease.
Of note, Allergan has scooped up licenses pertaining to both CRISPR/Cas9 and CRISPR/Cpf1. The latter was developed through the work of Feng Zhang and collaborators at the Broad Institute, MIT and Harvard — the institutions that Editas licenses its technology from. Both enzymatic approaches have distinct pros and cons, which means scientists would ideally have access to both.
Photo: Jay_Zynism, Getty Images