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

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.

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.

The U.S. Food and Drug Administration (FDA) announced the approval of Zynteglo (betibeglogene autotemcel), for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions. Ζynteglo the first cell-based one-time gene therapy product administered as a single dose. It is very important that Zynteglo is a customized treatment created using the patient’s bone marrow stem cells that are genetically modified to produce functional β-globin. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence, since they maintained a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. The most common adverse reactions associated with Zynteglo included reduced platelet and other blood cell levels. Also, there is a potential risk of blood cancer associated with this treatment therefore patients who receive Zynteglo should have their blood monitored for at least 15 years for any evidence of cancer. No cases of blood cancer have been seen in studies of Zynteglo. The FDA granted approval of Zynteglo to bluebird bio, Inc. More information can be found here.

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