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

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

The therapy, aimed at hereditary conditions like sickle cell anaemia and thalassaemia, was among the achievements discussed ...

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

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

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

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

Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously ...

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

Initially, it looked like the second transplant would work. Four days after the procedure, on Oct. 16, Saani was in front of the camera again. She wore pink pajamas and looked tired, but as ...

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

The biotech group struggled as a whole in 2024, but analysts say the innovation that has always supported the biotech ...