The business announced an amended collaboration with Celgene (NASDAQ:CELG) for developing engineered immune cells and will receive an upfront payment of $70 million as a result of the new agreement. The company also enjoyed a bump from peer CRISPR Therapeutics, which reported promising results for the first two patients dosed with its lead drug candidate, CTX001. Investors took that as evidence that CRISPR-based medicines might be the real deal, although that's a mighty big leap.
The gene editing company also reported a business update and operating results for the third quarter of 2019, but there wasn't much to report for the pre-commercial entity.
Editas Medicine started working with Juno Therapeutics, now owned by Celgene, in 2015. The idea was to combine the gene-editing platform of the former with the immunotherapy leadership of the latter. That's still the case, but the amended agreement scales back the specific types of engineered T cells that will be developed in the collaboration. It's a subtle, but potentially important, detail with (beneficial) ramifications for the long-term future of Editas Medicine.
It appears that the $70 million upfront payment was made in part to compensate Editas Medicine for the difference. After all, the company had already received $70 million in upfront, milestone, and execution payments under the original collaboration agreement. It's not immediately clear how the financial terms have changed, if they did at all, but the gene editing pioneer originally stood to receive up to $920 million in milestone payments.
Beyond that, there were several other updates provided in November:
- CRISPR Therapeutics reported interim results from the first two patients, one with sickle cell disease and one with transfusion dependent beta-thalassemia, dosed with its lead drug candidate. The promising results excited investors about the potential for CRISPR-based medicines and sent shares of gene editing peers soaring as well, but Editas Medicine relies on an entirely different approach. Therefore, investors got a little ahead of themselves.
- Pipeline updates are coming soon for Editas Medicine, though. The lead drug candidate, EDIT-101, will begin dosing patients in early 2020.
- The company also expects to report pre-clinical data for EDIT-301, being developed for the same blood disorders as CTX001, before the end of 2019. All things being equal, if it can compete with CTX001 on important metrics, then the drug candidate from Editas Medicine will be vastly more convenient and potentially lower-cost for patients.
The gene-editing landscape is still in the earliest stages of development. While CRISPR Therapeutics has taken an early lead as the top gene editing company, Editas Medicine is hoping to prove that its direct delivery approach will prove equally effective. The trial results the company will present in the coming years will become crucial tests for the future of CRISPR-gene editing, especially with competing techniques on the horizon.