Author Archives: Marta Figueiredo PhD

Blood Vessel Narrowing, Reduced Flow May Drive SCD Pain Crises

Blood vessel narrowing due to external or internal stimuli, and the resulting reduced blood flow, may be the main drivers of the painful vaso-occlusive crises experienced by people with sickle cell disease (SCD). Inducing stimuli for such blood vessel narrowing include mental stress, pain, and extreme temperatures, either low…

Oral Oxbryta Safe, Effective Over Long Term and in Real World

Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults with sickle cell disease (SCD). That’s according to nearly three years of data from the Phase 3 HOPE…

Ferriprox Approved in Brazil to Treat Iron Overload in SCD Patients

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use was also approved in combination with other iron-binding, or iron-chelating, treatments for people with thalassemia major, a severe…

UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial

The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood…

CTX001 Continues to Show Promise in Severe SCD

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.