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

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

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.

If we assume all patients who began treatment with Casgevy last year progress to the administration stage, CRISPR and Vertex ...

Orna will receive a $65m upfront payment and is eligible to receive potential milestone payments and royalties.

The campaign aims to reduce the spread of hereditary blood disorders such as sickle cell anaemia and beta-thalassemia ...

The "CRISPR-based Gene Editing Market by Product & Service, by Application, by End-User, and By Region" report has been added ...

Ongoing launch of CASGEVY® continues to gain momentum- -2025 is poised to be a catalyst-rich year with key updates across ...

The deal is set to help Vertex’s development of next-generation gene editing therapies for sickle cell disease and ...