Vertex, CRISPR's gene-editing treatment promising in early data by Amirah Al Idrus Wednesday, November 20, 2019 The data, from two patients with severe blood disorders, are "promising” for what could be a one-and-done treatment, Cantor Fitzgerald analysts wrote.
Disc Medicine nabs cash, big backers and biopharma deal by Ben Adams Tuesday, October 29, 2019 Disc Medicine got off a $50 million series A, led by Novo Nordisk’s VC arm, gained a new executive chair and a biopharma deal.
Novo taps bluebird bio for gene-editing tech by Ben Adams Wednesday, October 9, 2019 Looking to further broaden its reach outside of diabetes, Novo Nordisk has teamed up with bluebird bio to treat a series of genetic diseases.
FDA approves Roche's first test for whole blood donor screening by Conor Hale Friday, September 20, 2019 The FDA approved Roche’s first test for screening whole blood donations to detect a tick-transmitted parasite that lives in red blood cells.
Reversing sickle cell disease with CRISPR-edited stem cells by Angus Liu Wednesday, July 31, 2019 Fred Hutch scientists used CRISPR-Cas9 to boost the production of fetal hemoglobin by targeting a specific population of stem cells.
Editas, Allergan kick off long-awaited in vivo CRISPR trial by Amirah Al Idrus Friday, July 26, 2019 Though Editas and Allergan are not the first to test a CRISPR-based medicine in humans, their treatment is the first that edits DNA within the body.
UniQure weighs sale after hemophilia gene therapy turnaround by Nick Paul Taylor Monday, June 17, 2019 UniQure is reportedly working with advisers to assess options including a sale or partnership but is yet to decide on which path to take.
GBT sickle cell trial hits endpoint ahead of NDA filing by Nick Paul Taylor Friday, June 14, 2019 The proportion of patients who had a greater than 1 g/dL increase in hemoglobin is down on GBT’s prior update but still superior to placebo.
Celgene wraps up EU, U.S. filings for anemia drug luspatercept by Amirah Al Idrus Friday, April 26, 2019 Three weeks after filing luspatercept for FDA approval, Celgene has submitted the anemia drug for consideration by the European Medicines Agency.
Sangamo shares jump on gene, cell therapy updates by Ben Adams Tuesday, April 2, 2019 Sangamo saw its stock on the up this morning after it posted updates to a series of its trials using a new form of gene editing and cell therapies.