Topic: sickle cell anemia
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.
Cydan co-founders, CEO Chris Adams and R&D chief James McArthur discuss their hunt for orphan drugs.
Following its $15 million series A round earlier this year, BillionToOne has launched its first product for commercial and clinical use.
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.
A Fred Hutchinson team developed gold nanoparticles that can deliver CRISPR to mouse models of HIV and inherited blood disorders.
Sickle cell disease biotech Imara has nabbed Pfizer’s clinical rare disease lead Willem H. Scheele, M.D., as its new chief medical officer.