Vertex Pharmaceuticals expanded the company’s collaborative partnership with CRISPR Therapeutics to develop and commercialize a possible cure for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The focus is on the development of CTX001, an autologous, ex vivo CRISPR-CAS9 gene-edited therapy.
At the 2021 virtual JP Morgan Healthcare Conference, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”
Cambridge-based Magenta Therapeutics entered a non-exclusive research and clinical collaboration agreement with Beam Therapeutics.
South San Francisco-based Global Blood Therapeutics struck a deal with Syros Pharmaceuticals that is worth up to $375 million to discover, develop and commercialize novel therapies for the two types of blood disorders.
Bristol-Myers Squibb announced the completed acquisition of Celgene following the receipt of regulatory approval from all government authorities required by the merger agreement and, as announced in April 2019, approval by the companies’ stockholders.
CRISPR Therapeutics and Vertex Pharmaceuticals announced positive interim data from the first patients in the companies’ Phase I/II clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001.
The U.S. FDA approved Celgene and Acceleron Pharma’s Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
A European Medicines Agency panel recommended a conditional marketing approval for a gene therapy from Bluebird Bio Inc. as a genetic blood disorder treatment, setting the stage for the U.S. biotech to win the company’s first regulatory nod.
Celgene Corporation and Acceleron Pharma Inc. indicated the companies’ Phase III clinical trial of luspatercept in beta-thalassemia hit its primary endpoint of erythroid response.
Cambridge, Mass.-based bluebird bio’s LentiGlobin gene therapy is increasing hemoglobin levels in anemia patients battling sickle cell disease and beta thalassemia, which reduces the need for blood transfusions in those patients.