Author Archives: Marta Figueiredo PhD

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.

FDA Expands Approvals of Ferriprox

The U.S. Food and Drug Administration (FDA) has expanded the label of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to include patients ages 3 and older who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The therapy was first approved…