- Editas Medicine on May 15 disclosed during a first quarter earnings presentation that its highly anticipated CRISPR gene-editing therapy would be delayed entering the clinic.
- The biotech, which was seen as an early leader in the space, had expected to enter human trials for a rare eye disorder before the end of the year. The Investigative New Drug filing and subsequent study are now expected by mid-2018.
- According to Editas CEO Katrine Bosley, the setback for its LCA10 program is due to delays at a third-party manufacturer that produces the material for the adeno-associated viral (AAV) vectors used by Editas.
While the CRISPR field is still very much in its infancy, the space has been closely watched because of the potential the gene-editing technology could hold for revolutionizing medicine.
Editas has been considered the furthest along among the handful of CRISPR-focused companies racing to get a candidate into clinical trials. Rival CRISPR Therapeutics expects to file its clinical trial authorization for its lead beta-thalassemia program in Europe by the end of 2017, while Intellia Therapeutics is moving forward to IND-enabling studies later this year or early next.
"Now we realize this is a shift, but we do think it's the right decision for the program. The manufacturing delay related to production of input materials for AAV manufacturing. This caused us to have to shift to a latest slot at our AAV contract manufacturing site and collectively this pushed out the timeline," said Editas' Bosley during a first quarter call with investors.
"Importantly, there were no CRISPR-specific issues that caused this delay. And although no delay is ever a good thing, we understand that this is an important event. It does create a window opportunity to corporate elements of Allergan’s ophthalmology pre-clinical development and manufacturing expertise into the program," she added.
The Editas program in question is actually partnered with specialty pharma Allergan and is meant to treat Leber congenital amaurosis type 10, an eye disorder that primarily affects the retina. The treatment is part of a larger deal between the two companies.
The two teamed up during the first quarter to develop and commercialize CRISPR treatments for serious ocular diseases.
Allergan paid $90 million upfront, giving it the right to license up to five candidates, including LCA10. Allergan will develop and commercialize any products that it licenses, while Editas has the right to co-develop and co-promote two of the products. Editas is also eligible to receive $200 million in milestone payments per product that Allergan opts into.
While the IND filing might set back Editas slightly, the field is still so early that virtually anything can happen. These drugs are years away from reaching regulatory approval decisions and the rate at which they move through clinical trials could vary widely.