Topic: blood diseases

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 got off a $50 million series A, led by Novo Nordisk’s VC arm, gained a new executive chair and a biopharma deal.

Looking to further broaden its reach outside of diabetes, Novo Nordisk has teamed up with bluebird bio to treat a series of genetic diseases.

The FDA approved Roche’s first test for screening whole blood donations to detect a tick-transmitted parasite that lives in red blood cells.

Fred Hutch scientists used CRISPR-Cas9 to boost the production of fetal hemoglobin by targeting a specific population of stem cells.

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 is reportedly working with advisers to assess options including a sale or partnership but is yet to decide on which path to take. 

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.

Three weeks after filing luspatercept for FDA approval, Celgene has submitted the anemia drug for consideration by the European Medicines Agency.

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.