Boston based biotechnology company Vertex pharmaceuticals has entered into revised agreement with CRISPR Therapeutics to lead worldwide development, manufacturing and commercialization of gene editing therapy for sickle cell disease and beta thalassemia, CTX001. Under the terms of agreement, Vertex will pay USD 900 Mn upfront amount to CRISPR and an additional USD 200 Mn on approval of CTX001. Vertex will be responsible for 60% of program costs and receive the same percentage of profits form the worldwide sales of CTX001.
In 2015, the companies entered into strategic research collaboration focused on the use of CRISPR’s gene editing technology, with USD 75 Mn upfront payment from Vertex. CTX001 represents the first gene-based treatment that Vertex exclusively licensed from CRISPR. Currently undergoing Phase I/II clinical trials, the therapy is assessed for its efficacy and effectiveness in the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). The therapy has been granted a number of designations from the FDA and European regulators for sickle cell disease and beta thalassemia, including Regenerative Medicine Advanced Therapy, fast-track and orphan-drug tags. Previously, Bluebird Bio won approval in Europe for a β thalassemia therapy, but the firm has halted sales while it investigates two people in its sickle cell trials who developed cancer and CTX001 now remains a forerunner among gene-editing approaches being investigated for sickle cell disease and beta thalassemia.
Apart from the gene editing technology, Vertex pharmaceuticals is working on creating medicines for serious and rare diseases. Its portfolio comprises of commercially available therapy for the management of cystic fibrosis (CF) and has a robust pipeline of investigational small molecule medicines in other serious diseases including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases, sickle cell disease, beta thalassemia etc. CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. This collaboration will allow Vertex to use its robust R&D, manufacturing and commercialization expertise while supporting CRISPR in the development and innovation of CTX001 and indicative of the gaining momentum for development of gene-based therapies.