Thalassaemia International Federation (TIF) has published the 4th Edition 2021 Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT) in collaboration with editors Cappellini MD., Farmakis D., Porter J., and Taher A.  The TIF Guidelines are adopted and used extensively by academics, researchers and healthcare professionals all over the world as the only evidence-based reference text concerning the treatment of patients with TDT. The newly launched edition includes brand new chapters on the recently approved modalities of patient treatment, the value of patient engagement at the decision-making level, the Reference Centres’ contribution to patient care, and much more. You can access the publication here.

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), presented positive results from its Phase 2 proof-of-concept study of the oral pyruvate kinase (PK) activator "mitapivat" for non-transfusion-dependent thalassaemia (NTDT). This is the first clinical study of a PK activator in thalassaemia and the first drug trial in α-thalassaemia. The Phase 2 trial of mitapivat evaluated its efficacy, safety, pharmacokinetics and pharmacodynamics in 20 adult patients with either α- or β-NTDT and ≤10 g/dL baseline haemoglobin (Hb) levels. A sustained increase in Hb levels ≥1.0 g/dL from baseline was reported in 80% of the treated patients, together with improvements in hemolysis and ineffective erythropoiesis, and mild adverse drug reactions similar to previous studies in healthy volunteers and patients with PK deficiency. Agios plans to initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassaemia. More info: Agios press release

Bluebird bio announced that based on the analyses, it is very unlikely the Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML) reported in its Phase 1/2 (HGB-206) study of LentiGlobin gene therapy for sickle cell disease (SCD) was related to the BB305 lentiviral vector (LVV). Chief scientific officer reported that the integration site for the vector identified within the gene VAMP4, fact have confirmed by multiple independent analyses. VAMP4, has no known association with the development of AML nor with processes such as cellular proliferation or genome stability and also the insertion into the VAMP4 gene has had no impact on gene expression or gene regulation nor caused any disruption of nearby genes. Given this, the company has initiated engagement with regulators to begin the process of resuming clinical studies for sickle cell disease and β-thalassemia. More info: Bluebird bio press release.

Bluebird Bio is set to resume trials of LentiGlobin for sickle cell disease (SCD) and beta-thalassaemia after the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for SCD gene therapy (bb1111) for adult and pediatric patients with SCD, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies of betibeglogene autotemcel gene therapy (beti-cel; licensed as ZYNTEGLO™ in the EU and the UK) for adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT). The company is working closely with study investigators and clinical trial sites to resume all study activities as soon as possible. For more information, please see press release.

Bluebird bio announced the temporary suspension of its clinical studies of LentiGlobin gene therapy (autologous CD34+ cells encoding βA-T87Q-globin gene) for sickle cell disease (SCD) (bb111) after receiving reports of a suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML). This is the second patient with SCD, also treated with LentiGlobin, to be diagnosed with AML in a short period of time. These reports are currently under investigation and no link to the LentiGlobin gene therapy has been yet established. Although no cases of hematologic malignancy have been reported in any patient treated with the LentiGlobin gene therapy for transfusion-dependent β-thalassaemia (licensed as ZYNTEGLO in the European Union and the United Kingdom), but since it is the same BB305 lentiviral vector used in the LentiGlobin gene therapy for SCD, the company also temporarily suspended marketing of its authorized gene therapy ZYNTEGLO while the AML case is assessed. More info: Bluebird bio press release

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