Chow is City of Hope’s first sickle cell patient, while a beta thalassemia patient has been treated at Children’s National. Several authorized centers told WIRED they will begin infusions of ...
Some types of sickle cell disease cause severe illness, while others have a mild or unapparent course. Sickle cell disease is present from birth and inherited from genes you receive from your parents.
Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...
FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and ...
Orna Therapeutics and Vertex will use lipid nanoparticle system to develop gene-editing therapies for people with SCD and TDT ...
The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...
Achieved in vivo preclinical proof of concept of editing hematopoietic stem cells in non-human primates as a key step toward developing a novel ...
Vertex Pharmaceuticals Inc. will work with a Watertown biotech on new gene-editing therapies for people with sickle cell disease and transfusion-dependent beta thalassemia.
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