- Vertex Pharmaceuticals Inc. has selected CTX001 as the first drug it will co-develop and commercialize with CRISPR Therapeutics AG per the companies' ongoing collaboration.
- Executives have already requested from regulators to conduct a Phase 1/2 trial in Europe that would evaluate the therapy in adults with beta-thalassemia. Moving forward, Vertex and CRISPR Therapeutics plan on applying for another Phase 1/2 within the U.S. that would focus on CTX001 as a treatment for patients with sickle cell disease. The companies are aiming to get both studies underway in 2018.
- CTX001 is a preclinical, ex vivo therapy created with the eponymous CRISPR/Cas9 technology. Through a research deal inked in October 2015, Vertex gained the exclusive right to license as many as six targets from CRISPR Therapeutics.
Vertex currently reins over the cystic fibrosis market. Its investigational triple combination therapies have been highly effective in the clinic, and estimates hold the company could treat around 90% of the cystic fibrosis population within the next few years.
Yet, that dominance has also spurred investors and analysts to question what will be Vertex's second act. There are several options on the table, too, as the biotech has collaborations going with Parion Sciences Inc. for pulmonary disease treatments, Moderna Therapeutics Inc. for messenger RNA therapies and BioAxoe Biosciences Inc. for a spinal chord injury drug.
Gene-editing therapies, however, have been a solid guess — not only because they could help Vertex reach the small portion of patients that don't respond to its marketed products or won't respond to its combo regimens, but because of its partnership with CRISPR Therapeutics. By selecting the first drug to focus on within that partnership, Vertex reaffirms its interest in the gene therapy space.
"Over the past two years, we've made significant progress with CRISPR Therapeutics on the discovery and preclinical development of multiple CRISPR/Cas9-based treatments, and we're pleased to select CTX001 as the first of these treatments to move into clinical development as part of our collaboration," David Altshuler, Vertex's chief scientific officer, said in a Dec. 12 statement. "The addition of CTX001 to our clinical development pipeline provides us with a near-term opportunity to generate the first proof-of-concept clinical data for a CRISPR/Cas9-based medicine in two genetic diseases that are highly aligned with our research strategy."
CRISPR Therapeutics' also benefits from the selection, as it further validates the company's platform and offers the potential for profits down the road. For each non-hemoglobinapathy target, CRISPR Therapeutics could receive up to $420 million in milestone payments as well as royalties on any net product sales.
Also benefiting CRISPR Therapeutics is the fact that Vertex agreed to pay for all discovery research per the 2015 deal, and then split equally the development costs for potential hemoglobinapathy treatments. The biotech also agreed to a $75 million upfront payment and a $30 million investment in CRISPR Therapeutics.