News

SCD patients can access Casgevy via England’s public health system

Vertex Pharmaceuticals has entered a reimbursement agreement with the National Health Service in England that will enable eligible sickle cell disease (SCD) patients in that country to get access to treatment with Casgevy (exagamglogene autotemcel) via the public health system. “[This] agreement means eligible sickle cell disease…

SCD gene-editing therapy Casgevy now being offered at CHLA

Children’s Hospital Los Angeles (CHLA) is now offering Casgevy (exagamglogene autotemcel), an approved gene-editing therapy for sickle cell disease (SCD). “This is a transformative time for Children’s Hospital Los Angeles and the pediatric community we serve,” Alan S. Wayne, MD, pediatrician-in-chief at CHLA, said in a press…

Orna, Vertex partner on gene-editing therapies for SCD, TDT

Orna Therapeutics is partnering with Vertex Pharmaceuticals to develop gene-editing therapies for people with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The three-year collaboration will leverage Orna’s proprietary lipid nanoparticle (LNP) delivery system. LNPs are tiny vesicles made up of fatty molecules that can be used…