Publications

  Update: 27 September 2019 Rapamycin, a drug widely used for almost 20 years to protect organ transplant patients, has been found to reduce, in mice, the build up of toxic proteins that destroy red blood cells in β-thalassaemia. Investigators are moving forward to design a small proof-of-concept clinical trial ...

  Update: 27 September 2019 DRIVE PK is an ongoing global, open-label, Phase 2, safety and efficacy study evaluating Mitapivat in adults with PK deficiency who do not receive regular transfusions. It is the first clinical trial in adults with PK deficiency. Mitapivat is an investigational, first-in-class, oral, small molecule ...

  An educational leaflet aiming to strengthen the knowledge and understanding of patients with thalassaemia and other haemoglobin disorders across the world on most aspects relevant to gene therapy. English

CRISPR gene editing (SCD)   Update: 27 September 2019 Enrollment in Phase 1/2 study of CTX001 in patients with severe SCD is ongoing. Based on the progression of the program, CRISPR Therapeutics expects to obtain preliminary safety and efficacy data in late 2019. The first patient has been treated in ...

  Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   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 ...

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: 27 September 2019 No update available   Update: 30 July 2019 Results announced during 24th Annual Congress of the European Hematology Association show that treatment with single and multiple oral doses of VIT-2763 were well tolerated in healthy subjects, decreasing the iron levels in circulation. A proof ...

  Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   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 ...

  Update: 27 September 2019 No update available   Update: 30 July 2019 Sandoz, a Novartis division focusing on biosimilars have enrolled the first patient in an intergrated Phase I/III study for the development of denosumab for use in osteoporosis. Sources: https://www.novartis.com/news/media-releases/sandoz-announces-first-patient-enrolled-clinical-study-proposed-biosimilar-denosumab-osteoporosis   Update: 30 May 2019 No update available. ...

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 27 September 2019 No update available   Update: 30 July 2019 The FDA has accepted the Biologics License Application (BLA) for luspatercept. The FDA has granted Priority Review to this BLA for the evaluation of the beta-thalassemia indication and set ...