Agios reported the results of the study on Wednesday, and they were mixed. Most patients didn’t experience much of a ...

Pyrukynd, which Agios sells for a rare type of anemia, missed one of its main objectives in a study seen as crucial for its ...

Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect ...

Agios Pharmaceuticals said on Wednesday its sickle cell disease drug met one of its two main goals in a late-stage study of patients aged 16 and older but failed to show a statistically significant ...

Agios reported mixed results from a Phase 3 study of its drug Pyrukynd in sickle cell, raising doubts about the drug's ...

The CRISPR Cas9 gene editing tool — the genetic scissors that won the 2020 Nobel Prize in Chemistry — is essentially based on ...

Jayden Wilsey was the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease after federal approval in 2024.

Agios Pharmaceuticals plans to share data from RISE UP with the Food and Drug Administration to support the approval of the sickle cell disease indication.

Shares of Agios Pharmaceuticals sank after the company said a phase three trial of its mitapivat drug to treat Sickle Cell Disease missed some of its endpoints. The stock fell 34% to $30 after closing ...

India has launched BIRSA 101, the nation’s first indigenous CRISPR-based gene therapy for Sickle Cell Disease, marking a ...

Launching the therapy, Dr. Jitendra Singh declared that India has officially begun its decisive march toward a Sickle ...