As the fairness of orphan drug exclusivities is debated in Congress, the U.S. Food and Drug Administration granted Orphan Drug designations to Editas Medicine and Neurocrine Biosciences.
Weeks after Imara announced plans to discontinue the development of tovinontrinein for sickle cell disease, beta-thalassemia and heart failure indications, the company culled 83 percent of its workforce.
Shares of bluebird bio fell in trading on March 7 after the company revealed its dire financial straits.
Three companies – Intellia Therapeutics, Editas Medicine and uniQure – are each anticipating a bright 2022 as the promises of gene therapy continue to be realized.
Vertex Pharmaceuticals and Arbor Biotechnologies announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbor’s proprietary CRISPR gene-editing technology for select diseases.
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.