Topic: sickle cell anemia
Genentech veteran Frank Lee landed at Forma Therapeutics as it aimed to go from an early-stage company to one knocking on the door of approval.
Global Blood Therapeutics aims to bring a “paradigm shift” to the treatment of sickle cell disease with the launch of Oxbryta.
Adakveo is the first targeted therapy for vaso-occlusive crises—the painful clumps of sickled red blood cells that can block the flow of oxygen to organs—in patients with sickle cell disease.
2019 might be described as something of a "red-letter year" for patients with sickle cell disease overall, with two new drug approvals and positive data from gene therapy trials, but GlycoMimetics and Pfizer missed out on the celebrations.
This year’s top 15 list has been drawn up to include trials whose failure we judge to have had material consequences on their sponsors and the patients for whom the drugs are being developed. The catalog includes multiple trials for aggressive brain cancer, Alzheimer’s disease, nonalcoholic steatohepatitis and heart disease.
The data, from two patients with severe blood disorders, are "promising” for what could be a one-and-done treatment, Cantor Fitzgerald analysts wrote.
The goal is to start testing in vivo gene-based treatments for the diseases in humans within 10 years.
Trucode will use the money to advance gene editing assets designed to correct the mutations behind diseases including cystic fibrosis.
Sneaking in the bad news after hours on a Friday (a Bio-Twitter bugbear) still wasn’t under the radar enough to help stop the hemorrhaging of GlycoMimetics’ shares.
Fred Hutch scientists used CRISPR-Cas9 to boost the production of fetal hemoglobin by targeting a specific population of stem cells.