Editas bags $25M from Allergan as FDA signs off on human CRISPR trial

CRISPR technology for genome editing (Source: AstraZeneca)
Editas' LCA10 treatment is slated to be the first in vivo CRISPR treatment tested in humans. (AstraZeneca)

After a year’s delay, Editas Medicine is finally back on track. The gene-editing biotech announced Friday the FDA has OK’d its IND application and that it will start enrolling patients in a phase 1/2 trial of its CRISPR-based treatment for LCA10, a rare form of blindness. Editas collected a $25 million milestone payment from Allergan. 

Editas once expected to file the IND for its Allergan-partnered treatment, EDIT-101, by the end of 2017. But a manufacturing problem disclosed in May that year delayed those plans, allowing CRISPR Therapeutics and partner Vertex to get the jump on Editas. They kicked off a European trial of their ex vivo CRISPR-based treatment for beta thalassemia in August. But even Vertex and CRISPR have not been trouble-free—the FDA placed a clinical hold on their treatment, CTX001, in May, putting off their plans to start a clinical trial in sickle cell disease in the U.S. The agency lifted the hold in October. 

EDIT-101 is in development for LCA10, a form of Leber congenital amaurosis that is caused by a p.Cys998X mutation in the CEP290 gene. The setback came from a misstep in the production of a material used to make the adeno-associated viral vectors Editas will use to deliver its gene-editing treatment. 

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“AAV manufacturing requires several steps to happen in perfect sequence for things to all come together. And we’re using several external contractors to perform these steps. We have to produce the input material that all comes together to then create the AAV in a cell culture systems,” Vic Meyer, Editas’ chief technology officer, told investors at the time. 

“In our case, one of the input materials failed a quality specification and we needed to go back and remake that material. That delay in remaking the material caused us to miss the manufacturing slot with the AAV CMO and that combined with the remaking material pushed out the timeline.” 

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Although Vertex and CRISPR have nabbed the distinction of running the first company-backed CRISPR trial, Editas may yet get a first under its belt. EDIT-101 is slated to be the first in vivo CRISPR treatment tested in humans—that is, the gene editing takes place inside the body. Vertex and CRISPR’s beta thalassemia treatment, on the other hand, involves ex vivo gene editing—a patient’s cells are harvested and edited to increase fetal hemoglobin levels in the patient’s blood cells. The edited cells are then infused back into the patient where they are expected to produce blood cells with fetal hemoglobin and compensate for defective adult hemoglobin.