A deal-hungry Vertex ushers in three more gene editing programs as it closes out harbinger CRISPR deal
Back in 2015, Vertex made a splash in the nascent CRISPR gene editing world by wagering $105 million to partner with one of its pioneers — then-private startup CRISPR Therapeutics — to generate up to six new treatments for genetic diseases.
Fast forward four years, the duo has accomplished the historic feat of dosing their ex vivo therapy, CTX001, in patients with beta thalassemia, and Vertex has clearly signaled its intent on going bigger into gene editing tech. As they wrap up the research part of their collaboration, the big biotech is tapping three more CRISPR programs for its pipeline, which has ballooned in recent weeks with new drugs introduced from new pacts.
In a brief update tucked into its Q3 report, Switzerland-based CRISPR Therapeutics noted that Vertex has exercised its options to license three additional targets, including the cystic fibrosis transmembrane conductance regulator (CFTR) gene and two others. That triggered a $30 million upfront payment — $10 million each — and each project can earn as much as $410 million in milestones.
Mutations in the CFTR gene is the culprit for cystic fibrosis, a serious disease characterized by the buildup of thick mucus in the lungs. Vertex’s portfolio of approved therapies, including the latest breakthrough triplet Trikafta, work by tinkering with the resulting mutant proteins.
The CRISPR approach, on the other hand, aims to repair the defects (and there are hundreds of variations) at their roots.
In all Vertex has exercised options on five programs, a spokesperson confirmed: In addition to beta thalassemia, they are also testing CTX-001 in sickle cell disease. Patient enrollment is ongoing for both Phase I trials, and CRISPR remains involved in the development.
And they are not done yet. In June Vertex unveiled a $245 million acquisition of Exonics, UT Southwestern investigator Eric Olson’s gene-editing startup laser-focused on Duchenne muscular dystrophy, and expanded its alliance with CRISPR Therapeutics to cover DMD as well as myotonic dystrophy type 1. The expansion cost $175 million upfront.
Those deals were the harbinger of a shopping spree in which Vertex also picked up a stem-cell-based potential cure for Type 1 diabetes and secured access to a small molecule platform for drugging RNA — all before CEO Jeff Leiden is set to hand the baton to CMO Reshma Kewalramani, moving to the chairman post.
Now that Vertex has essentially conquered CF — both on the regulatory and reimbursement fronts, having finally struck a deal with England’s health system — Kewalramani’s tenure will be defined by the new technologies and disease areas that she chooses to prioritize.
She will be doing so with a top team that has a balanced gender mix, following the promotions of Carmen Bozic, formerly EVP of global medicines development and medical affairs, to CMO and Nia Tatsis to head of regulatory.