With $19 million in seed funding led by the Retinal Degeneration Fund, Opus Genetics launched with a mission to develop gene therapies for rare forms of blindness driven by genetic mutations.
Shares of bluebird bio climbed in trading after the company announced on March 10 that analyses demonstrate the lentiviral vector BB305 was unlikely to be the cause of acute myeloid leukemia (AML) reported in an early-stage clinical study of LentiGlobin for sickle cell disease.
Alnylam Pharmaceuticals presented complete results from the company’s ILLUMINATE-B Phase III study in children under 6 years with primary hyperoxaluria type 1 (PH1), a rare disorder affecting the kidneys.
An experimental Amgen Inc. drug that targets a specific genetic mutation shrank tumors in 32% of advanced lung cancer patients and 7% of those with colon cancer, according to data from an early-stage trial.
Researchers at the The Scripps Research Institute found that the strains spreading so quickly in Europe and the U.S. have a mutated S “spike” protein that makes it about 10 times more infectious than the strain that originally was identified in Asia.
The U.S. Food and Drug Administration approved Clovis Oncology Inc.’s drug Rubraca for the treatment of adult patients with metastatic castration-resistant prostate cancer, whose tumors have a genetic mutation.
The first treatment for adult patients with metastatic non-small cell lung cancer (NSCLC) who also have a specific genetic mutation was approved by the U.S. Food and Drug Administration.
Germany’s drug assessment body said data provided by Bayer on the precision cancer drug Vitrakvi did not provide clear enough evidence of benefits, in a setback to the drugmaker in the company’s home market.
Merck & Co. Inc. is buying ArQule Inc. for $2.7 billion, seeking to tap into the drug developer’s experimental blood cancer therapy that targets genetic mutations.
Novartis AG won U.S. approval for the company’s experimental sickle cell disease drug Adakveo.