Vertex develops first-ever approved gene-editing treatment for sickle cell disease
New England Council member, in partnership with CRISPR Therapeutics, have achieved a pivotal milestone with the U.S. Food and Drug Administration’s (FDA) approval of CASGEVY™ sickle cell disease (SCD) treatment.
SCD is an inherited blood disorder characterized by abnormally shaped red blood cells, causing blockages in blood vessels and resulting in severe pain, organ damage, and a shortened lifespan. This groundbreaking therapy targets recurrent vaso-occlusive crises (VOCs) in SCD patients aged 12 and above. By utilizing CRISPR/Cas9 technology to edit hematopoietic stem cells, CASGEVY aims to reduce BCL11A expression and increase fetal hemoglobin (HbF) production, offering the potential for a durable, one-time treatment to address severe VOCs and related hospitalizations. The treatment works like molecular scissors to change certain cells in the body, aiming to reduce severe pain events and the need for frequent hospital visits in patients with SCD. With its ongoing evaluation for broader applications, including transfusion-dependent beta-thalassemia (TDT), this innovative therapy presents a new possibility for individuals grappling with the challenges of SCD and related conditions.
“CASGEVY’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the U.S. As importantly, CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “I want to convey my deepest gratitude to the patients and investigators whose trust in this program paved the way for this landmark approval.”
The New England Council would like to congratulate Vertex on developing this groundbreaking treatment.
Read more in the Boston Business Journal.