Publications

CRISPR gene editing (SCD)   Update: 30 May 2019 No update available.   Update: 29 March 2019 The Phase 1/2 Study in Sickle Cell Disease (SCD) is an open-label trial aiming to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with ...

  Update: 30 May 2019  Apotransferrinhas a physiological role in the transportation and distribution of iron among the body organs. Apotrasferrin has received orphan drug designation from EMA A proof of concept study in the Netherlands will begin in the second half of 2019 lasting 16 weeks with a maximum ...

An educational leaflet with important information for β-thalassaemia patients on hematopoietic stem cell transplantation (HSCT), more commonly known as bone marrow transplantation (BMT). English

VIT-2763   Update: 30 May 2019 No update available.    Update: 01 February 2019 A randomized, controlled, multi-centred phase 2 proof-of-concept trial will begin in the second half of 2019 to assess the effectiveness of the oral ferroportin inhibitor (VIT-2763) on iron overload in β-thalassaemia patients. Phase 1 trials have ...

  Update: 30 May 2019 No update available.   Update: 29 March 2019 SLN124 has been submitted to the UK Medicines and Healthcare Products Regulatory Agency (MHRA). Subject to approval from the MHRA, Silence intends to begin a Phase I/II First-in-Human study in ß -Thalassemia and MDS patients, with the ...

  Update: 30 May 2019 No update available.   Update: 29 March 2019 No update available.   Update: 01 February 2019 No update available.   Update: 20 December 2018 A single-site, placebo-controlled, double-blind phase 2b trial that included 63 patients with transfusion-dependent thalassaemia and bone mineral density T scores between ...

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 30 May 2019 Application for Marketing Authorization to the European Medicines Agency (EMA) for luspatercept for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions has been submitted.   Submission to the FDA Biologics License Application (BLA) ...

Assessment of long-term safety of deferiprone in children (DEEP-3 Study)     Update: 20 December 2018 The study has concluded.   Update: 02 October 2018 The safety of deferiprone, either alone or in combination with deferoxamine, was evaluated in 297 patients from 16 hospitals in 6 Mediterranean countries, who had ...

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 30 May 2019 Upon receipt of marketing authorisation from the European Commission by the end of June, Bluebird expects to launch first in Germany, followed by Italy, France and the U.K. in 2020 Analysts estimate prices of $900,000 in Europe ...

CRISPR gene editing   Update: 30 May 2019 The FDA has granted Fast Track Designation for CTX001 for the treatment of transfusion dependent beta thalassaemia. Sources: https://www.globenewswire.com/news-release/2019/04/16/1804601/0/en/CRISPR-Therapeutics-and-Vertex-Announce-FDA-Fast-Track-Designation-for-CTX001-for-the-Treatment-of-Beta-Thalassemia.html   Update: 29 March 2019 The Phase 1/2 Study in Beta Thalassaemia is an open-label trial aiming to assess the safety and efficacy ...