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

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

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.

It can generate billions in revenue from both acute and possibly chronic pain. If approved, it would give Vertex ...

Achieved in vivo preclinical proof of concept of editing hematopoietic stem cells in non-human primates as a key step toward developing a novel ...

Agios Pharma submitted a sNDA to the FDA after two Phase III trials studying PYRUKYND (mitapivat) in thalassemia met their ...

Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...

CRISPR stock decline is influenced by Editas Medicine's struggles, but CRSP remains strong with FDA approval and strategic ...

Vertex Pharmaceuticals (NASDAQ: VRTX) might be one of those special cases. Should you buy Vertex stock hand over fist before ...

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 ...

BRL-101 is under clinical development by BRL Medicine and currently in Phase II for Sickle Cell Disease. According to GlobalData, Phase II drugs for Sickle Cell Disease have a 59% phase transition ...

Their discovery may chart a path to a small-molecule drug that could be made available to all patients with sickle cell disease, as well as those with beta thalassemia. However, Casgevy and other ...