The majority of SCD patients given Lyfgenia in clinical trials remained free of vaso-occlusive events (VOEs) following ...

The most-read gene therapy articles included topics on FDA approvals, chimeric antigen receptor T-cell therapies, the ...

(BEAM) announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs). Updated data ...

Vertex Pharmaceuticals Incorporated looks set for two new drug approvals in early 2025. Click here to read an analysis of ...

Jefferies has upgraded Vertex Pharmaceuticals Inc. (NASDAQ:VRTX), citing the company’s robust cystic fibrosis base business ...

SAN DIEGO — The approval one year ago of two genetic medicines for sickle cell disease, including the first therapy powered by CRISPR gene editing, was a milestone for people living with a ...

Genetic Carriers for Sickle Cell Disease Have Higher Risks of Blood ... while activating a previously overlooked blood-clotting mechanism, stemming ... Use of Anticoagulant Drug After Aortic ...

Trials and Tribulations of Cell Therapy for ... the risk of cardiovascular disease (CVD) and coronary heart disease (CHD), according to a new study. ... Newfound Mechanism May Explain Why Some ...

Prof. Azem concludes, "Our study decodes the mechanism of a severe and rare neurological disease caused by a mutation in a protein critical for importing proteins into the mitochondria.

Advances in peptide drug development and the rising prevalence of hematological disorders are driving demand for peptide-based therapeu ...

Positive results from the pivotal LUNA 3 phase 3 study of rilzabrutinib in adults with persistent or chronic immune thrombocytopenia (ITP), a rare immune-mediated disease, reinforce the efficacy and ...

Through its research and development efforts, the company focuses on cystic fibrosis, sickle cell diseases, beta thalassemia, type-1 diabetes and pain. It uses state-of-the-art technology platforms to ...