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“Empowering Lives, Embracing Progress: Equitable and Accessible Thalassaemia Treatment for All’’, is the theme that Thalassaemia International Federation (TIF) announced for the International Thalassaemia Day (ITD) on 8 May 2024. The 2024 theme is a powerful call to raise awareness about this condition and its impact while celebrating the solidarity of the resilient thalassaemia community worldwide. Furthermore, the theme champions the empowerment of people living with thalassaemia by leveraging advances in treatment options and highlighting our shared responsibility to raise awareness and advocate tirelessly for meaningful, positive change. The focus of TIF for 2024 is unambiguous: to ensure all individuals with thalassaemia, no matter their location or economic circumstance, receive access to accurate diagnosis, current and future treatments, and well-rounded care. More information about theme can be found here.

Editas Medicine, Inc. announced positive initial clinical data from the first two patients with sickle cell disease (SCD) treated with EDIT-301 in the Phase 1/2 RUBY trial. EDIT-301 is an investigational medicine supported by the Company's proprietary engineered AsCas12a enzyme to generate high efficiency edits in the HBG1/2 promoter site and disrupt the binding site of BCL11a, consistent with observed naturally occurring human mutations that lead to high levels of fetal hemoglobin. The clinical data includes safety data from the first two patients and efficacy data from the first patient treated. EDIT-301 was well-tolerated in the two patients and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. No serious adverse events occurred, and no adverse events reported were related to treatment with EDIT-301. For more information: press release

The ITHANET virtual information day was held with great success on September 29. The information day appealed to a wide audience that included researchers, doctors and academics from all over the world. More than 100 people from 28 countries registered for the event and over 50 participants attended all talks. The event covered recent enhancements to the ITHANET community portal for haemoglobinopathies and the international partnerships that were established through the project. Along with the established ITHANET databases, the newly developed IthaPhen and IthaCNVs tools were also presented in detail. The event also focused on studies using ITHANET data. To watch the full event follow this link using the passcode aIw8P?Bj.

Thalassaemia International Federation (TIF) has recently published its 2021 Annual Report that provides insight to the Federation’s numerous activities, educational and advocacy initiatives, events, and productive partnerships, while also highlighting key challenges and tools for the prioritisation of haemoglobinopathies on health agendas. Milestones include

  • Renewal of TIF's Official Relations with WHO
  • Publication of The Global Thalassaemia Review as part of TIF's collaboration with WHO
  • Publication of The Guidelines for the Clinical Management of Thalassaemia (4th edition)
  • New tools for national health authorities: A cost-of-illness estination model and disability stratification algorithm
  • The Panos and George Englezos Awards acknowledges the contribution of eminent patient advoates and healthcare professionals
  • THALassaemia In Action (THALIA) 2018-2021 project comes to and end

The 2021 Annual Report is available here for download.

We are pleased to announce the organization of the ITHANET virtual information day and invite you to attend on Sep 29th 14:00-17:00 GMT+3. During the event, the recent developments and collaborations of the ITHANET community portal for haemoglobinopathies will be presented. The event’s poster and agenda can be found here. Please register here by Sep 25th.

  1. FDA approved the First Cell-Based Gene Therapy for Adult and Pediatric Patients with Regular Blood Transfusions β-Thalassemia
  2. Pfizer will Acquire Global Blood Therapeutics for $5.4 Billion to further Enhance Its Role in Rare Hematology
  3. Editas: promises therapy with EDIT-301 in the first patient with Sickle Cell Disease
  4. Beti-cel Gene therapy for people with transfusion dependent β-thalassemia supported by FDA Advisory Committee

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