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CTX001™ (CRISPR Therapeutics and Vertex Pharmaceuticals) has received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). PRIME provides early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. CTX001™ is an investigational gene-editing cell therapy that uses the CRISPR-Cas9 gene-editing tool to modify patient-derived stem cells to express foetal haemoglobin (HbF). An increase in HbF levels has the potential to alleviate transfusion requirements for severe thalassaemia patients and reduce painful and debilitating sickle crises for SCD patients. CTX001 is currently the most advanced gene-editing approach in development for thalassaemia and SCD. To date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission, for both transfusion-dependent thalassaemia and SCD.
For more information about the clinical trials and the gene-editing process, see here.


The FDA granted Rare Pediatric Disease (RPD) designation for EDIT-301 (Editas Medicine, Inc.), an experimental, autologous cell medicine intended for the treatment of sickle cell disease (SCD). EDIT-301 comprises sickle patient haematopoietic stem/progenitor cells (HSPCs) that are genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the γ-globin gene promoter region in the beta-globin locus. Red blood cells (RBCs) derived from EDIT-301 HSPCs demonstrate a sustained increase in foetal haemoglobin (HbF) production, which inhibits polymerisation of sickle haemoglobin (HbS) in RBCs, a hallmark of SCD, and has the potential to provide long-term treatment benefits for people living with SCD. The Company expects to file to the FDA for Investigational New Drug (IND) for SCD by the end of 2020. Source: ASH News

Xromi® (Nova Laboratories Ltd) has been approved by the Scottish Medicines Consortium for use within NHS Scotland to treat sickle cell disease (SCD) in patients as young as 2 years of age. Xromi® is a strawberry-flavored oral formulation of hydroxycarbamide (hydroxyurea) intended for the prevention of vaso-occlusive complications in SCD patients who have difficulty swalling tablets. This is the first  hydroxycarbamide formulation appropriate for young children. Xromi® is available on a prescription-only basis to healthcare specialists in the UK, EU and the Middle East, with plans for worldwide availability.

See here for more info on Xromi®. Source: Sickle Cell Disease News

Supported by the American Society of Hematology (ASH), the first set of recommendations aimed at increasing capacity for specialized care for adults living with sickle cell disease (SCD) have recently been published in Blood Advances. SCD is a debilitating, lifelong disease with severe complications beginning early in life. Given improvements in care, most children with SCD survive into adulthood. Across the United States, surveys suggest significant gaps and dissatisfaction with adult sickle cell care for reasons such as difficulties with access, perceived discrimination, and lack of clinician confidence and knowledge in managing SCD complication. The proposed recommendations codify the components of establishing SCD adult care centers. These include:

  • Multidisciplinary, team-based, evidence-guided care that is coordinated throughout the institution.
  • The SCD center as the recognized authority for managing SCD within the institution.
  • A physician lead who is considered an SCD specialist.
  • One or more social workers, a patient coordinator, and dedicated nursing staff.
  • The ability to offer acute and chronic pain management, transfusion, and access to specialists.

The paper makes recommendations for additional important, but not required, personnel such as clinic managers, behavioral health staff and psychologists, physical and occupational therapists, and pharmacists. Altogether, the recommendations define comprehensive care for SCD as team-based, holistic, and tailored to the unique needs of individuals with SCD.

More information: Blood Advances, ASH news

The European Rare Blood Disorders Platform (ENROL) conceived in the core of ERN-EuroBloodNet to serve as an umbrella for both new and already existing registries on rare haematological disorders (RHD), has officially started June 1st, 2020. ENROL online kick-off meeting was successfully held last July 2nd with more than 120 registered participants, including ERN-EuroBloodNet members, affiliated partners, candidates, EURORDIS and patients’ representatives, European Hematology Association (EHA) representatives, researchers and registries’ curators. The meeting started with a welcome and overview of ENROL from the platform coordinator followed by two blocks of presentations highlighting ENROL implementation by the different tasks leaders and the involvement of the different stakeholders as fundamental pillars for the successful implementation of ENROL, including patients, hospitals’ CEOs and the legal and ethical teams. For more information, the main outcomes of the meeting and the slides presented are available here.

  1. FT-4202 receives FDA Fast Track designation for Sickle cell disease
  2. Imara Receives Orphan Drug Designation for IMR-687 for Treatment of β-Thalassemia
  3. European Commission approves REBLOZYL® for β-thalassaemia
  4. Agios Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent α- and β-Thalassemia in Phase 2 Study of Mitapivat Activator

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