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.

FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and ...

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...

Orna Therapeutics and Vertex will use lipid nanoparticle system to develop gene-editing therapies for people with SCD and TDT ...

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.

Editas Medicine (EDIT) announced its three-year strategic priorities, anticipated 2025 key milestones, and new in vivo preclinical proof of ...

Anticipated 2025 milestones include: declare two in vivo development candidates, one in HSCs and one in liver; present further in vivo HSC data; present in vivo data in one liver indication; establish ...