Vertex puts up $900 million to lead CRISPR-partnered gene editing programme

Vertex Pharmaceuticals will pay CRISPR Therapeutics $900 million upfront as part of an amendment to an existing partnership between the two that will give Vertex majority control over development and marketing of the experimental CRISPR/Cas9-based gene editing therapy CTX001, the companies said Tuesday. CTX001 is currently in studies evaluating its potential as a curative therapy for sickle-cell disease (SCD) and transfusion-dependent beta-thalassaemia (TDT).

As part of the amended deal, Vertex will take on 60% of the programme's costs and receive 60% of profits from future sales. Vertex had paid $105 million upfront under the initial 2015 collaboration, with the partners agreeing at the time to split R&D costs and future sales 50-50 for gene editing products to treat haemoglobinopathies. Under the updated deal, CRISPR Therapeutics will be responsible for 40% of the costs in return for 40% of profits, and it also stands to receive a $200-million milestone payment if CTX001 obtains regulatory approval.

'Sustained' response in TDT, SCD patients

"Cell and genetic therapies are key to our strategy," remarked Vertex executive chairman Jeffrey Leiden, adding "we see a significant commercial opportunity for CTX001." Leiden explained that the increased investment "is based on the compelling clinical profile of CTX001," as well as "the rapid progress that we and our partners…have made toward registration and commercialisation."

In December, the companies reported updated findings involving 10 patients in two Phase I/II trials of CTX001 who exhibited a "consistent and sustained response" to treatment at follow-ups ranging from three to up to 18 months. Specifically, the CLIMB-111 study showed that TDT patients remained transfusion-independent after infusion, while in the CLIMB-121 trial, patients with severe SCD remained free of vaso-occlusive crises. In both cases, participants achieved normal to near-normal haemoglobin levels.

New operating model

Samarth Kulkarni, chief executive at CRISPR Therapeutics, said "we have now dosed more than 30 patients with CTX001, with longest follow-up beyond two years, and we are on track to complete enrollment in both clinical trials this year." Kulkarni also indicated that "we are adopting a new operating model to enable a globally coordinated launch of CTX001, leveraging Vertex's…capabilities and leadership in development, manufacturing and commercialisation to enable this medicine to reach all patients that can benefit from it as quickly as possible."

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