CTX001™ (CRISPR Therapeutics and Vertex Pharmaceuticals) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease (SCD) and transfusion-dependent β-thalassaemia (TDT). CTX001™ is an investigational gene-editing cell therapy that uses the CRISPR-Cas9 gene-editing tool to modify patient-derived stem cells to express fetal haemoglobin (HbF). An increase in HbF levels can compensate for the abnormal synthesis and function of adult haemoglobin in adult patients with SCD and TDT, and ameliorate the clinical and hematologic severity of both disorders. Preliminary data from the Phase 1/2 CLIMB-SCD-121 trial (NCT03745287), assessing the safety and effectiveness of a single dose of CTX001™ in patients 18 to 35 years of age with SCD, has shown that the therapy safely increased HbF levels and effectively prevented the occurrence of vaso-occlusice crises. Positive early findings have also been reported for the first TDT patient receiving CTX001™ in the Phase 1/2 CLIMB-Thal-111 trial (NCT03655678), for which patients are still being recruited. In addition to RMAT designation, CTX001™ has received Orphan Drug Designation from the FDA for TDT and from the European Medicines Agency (EMA) for TDT and SCD. CTX001™ has also Fast Track Designation from the FDA for both disorders. For more information, see here.

The Committee for Medicinal Products for Human Use (CHMP) recommended the granting of a marketing authorization for the medicinal product Reblozyl, on the 30th of April 2020. Reblozyl is a novel erythroid maturation agent, intended to treat adult patients with β-thalassaemia who require regular red blood cell transfusions. Also, Reblozyl has been approved for the treatment of adult patients with myelodysplastic syndrome (MDS). Reblozyl has the potential to decrease the number of transfusions in patients with β-thalassaemia and MDS. The European Commission will review the recommendation for this medicinal product in order to approve or not the Reblozyl in the European Union. Authorized marketing of Reblozyl in the European Union will represent a new class of therapy for adult β-thalassaemia patients who have limited treatment options. For more information, see here.

ERN-EuroBloodNet has established a European collaborative platform for collecting information to quickly identify the impact of COVID-19 on patients with red blood cell disorders, especially sickle cell disease and thalassaemia, and to understand the impact of risk factors, including age and disease complications on the course and severity of COVID-19 infection. The proposed registry has been developed by Vall d'Hebron Research Institute using Redcap, a secure web application for building and managing online databases. The network of hospitals created from this registry will continuously analyse the information on the clinical management and outcomes in hematological patients and will collaboratively make recommendations for COVID-19 management in this group of patients. Additionally, this collaborative effort will support daily medical practice and enable inter-professional consultation of complex cases. Individual patients' data will be gathered in a codified way. For more information, see here.

International Thalassaemia Day is marked on May 8 every year to raise awareness among the general public and decision-makers about thalassaemia across the globe. This day commemorates the lifelong and difficult struggles of patients living with this inherited blood disorder to lead a better life. This day is observed by the Thalassaemia International Federation (TIF). World Thalassaemia Day theme 2020 is ''The dawning of a new era for thalassaemia: Time for a global effort to make novel therapies accessible and affordable to patients". For more info, see here.

The American Society of Hematology (ASH) has created a resource webpage in the midst of the global pandemic to provide public health professionals and the general public with haematology-specific up-to-date COVID-19 information in a series of Frequently Asked Questions (FAQs), as well as insights into COVID-19 treatment and several links to relevant resources, including research updates and health e-stats. Specifically, in the non-malignant category, ASH publishes FAQs on sickle cell disease (SCD, link) and thalassaemia (link) to help guide providers who are less familiar with these diseases to care and treat SCD and thalassaemia patients infected with COVID-19. All of the COVID-19 content is free to access. For more information, see here.