- Vertex Pharmaceuticals and CRISPR Therapeutics can start testing an experimental gene-editing therapy in U.S. patients with sickle cell disease now that the Food and Drug Administration has lifted a clinical hold instituted several months ago.
- The hold came in May, shortly after Vertex and CRISPR submitted an Investigational New Drug application seeking to evaluate CTX001 as a treatment for the blood disorder. The companies noted both in recent financial filings and in an Oct. 10 statement that regulators in "multiple countries" outside the U.S. have given them the go-ahead to start clinical trials of CTX001 in sickle cell disease as well as beta-thalassemia.
- With the hold resolved, the companies said they plan to initiate a Phase 1/2 study of CTX001 in sickle cell patients in the U.S. by year's end. Another Phase 1/2 study in Europe is currently enrolling, and will test the therapy in patients with transfusion dependent beta-thalassemia.
In spite of being very early-stage, CTX001 may be quite impactful to its developers.
For one, it serves as the lead candidate in CRISPR's pipeline. Showing the therapy is effective and safe in the clinic could not only strengthen the outlook for the company's technology platform — particularly regarding ex-vivo development — but also help alleviate some of the concerns hanging over gene-editing treatments.
Earlier this year, share value at companies like CRISPR, Intellia Theapeutics and Sangamo Therapeutics fell after two studies suggested the CRISPR/Cas9 system widely used for gene-editing works better in cells that are more likely to become cancerous. Those stock sell offs could be described as an overreaction, yet they nonetheless underscore how skittish investors can be when it comes to new tech.
Furthering that point, CRISPR shares were up more than 15% in after-hours trading Wednesday following the news of the clinical hold's lift.
Vertex, meanwhile, doesn't get as rattled by such worries, given it has three marketed cystic fibrosis drugs and a dominant position in that market.
Still, investors have already started to wonder what will be Vertex's next play. Gene-editing seems to be as good a bet as any, due to the biotech's partnership with CRISPR and the prediction it can treat the entire cystic fibrosis population if triple combinations and gene-focused therapies come into its portfolio.
On a second quarter earnings call, Vertex's Chief Operating Officer, Ian Smith, noted that early-stage tech was one of the three key areas on which the company was focusing.
"You've seen us complete a couple of deals in the last couple of years that have been very important for us in terms of getting us into new areas of science and new modalities," he said, later highlighting the collaboration with Vertex "where we've made really nice progresses in beta-thalassemia and sickle cell."
In addition to those two programs, Vertex also has an option to exclusively license from CRISPR a preclinical in vivo program for cystic fibrosis.