Vertex Pharmaceuticals has amended its agreement with CRISPR Therapeutics to take control of their gene therapy partnership.
Following the amended agreement, Vertex will now lead the global development, manufacturing and commercialisation of the gene therapy CTX001, with support from CRISPR Therapeutics.
In December, Vertex and CRISPR Therapeutics announced new, early-stage data from a total of ten patients treated with the CRISPR/Cas9 gene-editing therapy.
According to the companies, seven patients with transfusion-dependent beta thalassaemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at the last follow-up.
A further three patients with sickle cell disease (SCD) treated with CTX001 were free of vaso-occlusive (VOCs) until the last follow-up.
Currently, CTX001 is being investigated in two ongoing phase 1/2 clinical trials, as a potential one-time treatment for patients with TDT and severe SCD.
“Cell and genetic therapies are key to our strategy of developing transformative therapies for serious diseases, and this agreement is an important next step in cementing our leadership in these modalities as we bring forward our broad gene and cell-based therapeutics portfolio,” said Jeffrey Leiden, executive chairman of Vertex.
“Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialisation,” he added.
Under the terms of the amended agreement, Vertex will be responsible for 60% of programme costs for CTX001 and will receive 60% of profits from future sales of the therapy worldwide.
This represents a 10% increase in programme economics, compared to the original agreement.
CRISPR will also receive a $900m upfront payment, with further potential for a $200m payment upon the first regulatory approval of CTX001.
Along with Vertex/CRISPR Therapeutics, bluebird bio is also seeking to bring its gene therapy LentiGlobin for SCD (bb1111) to market.