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.
Brisbane, California-based Sangamo Therapeutics announced that Sanofi US was returning rights to SAR445136 as the Paris-based company shifts its approach from personalized cell therapies to allogeneic off-the-shelf genomic approaches.
Shares of Global Blood Therapeutics were up more than 10 percent in trading on December 17 following regulatory approval of a supplemental New Drug Application for oral Oxbryta (voxelotor) tablets for the treatment of pediatric sickle cell disease (SCD).
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.
In a countersuit, Cambridge, Mass.-based bluebird bio is hitting back at Roche-owned Spark Therapeutics over the use of the word “spark” in promotional campaigns involving the company’s “Be the Spark” campaign against sickle cell disease.
In a flurry of activity, multiple biopharma companies made their first appearance on the Nasdaq Stock Exchange on June 25, raising hundreds of millions of dollars to advance the development of next-generation therapeutics and scale their businesses.
The U.S. Food and Drug Administration approved Chiesi Global Rare Diseases’ Ferriprox (deferiprone) for the treatment of transfusional iron overload caused by sickle cell disease or other anemias in adults and children ages 3 years and older.
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.