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Clinical trial updates

Rapamycin

  Update: 25 November 2019 No update available.   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 ...

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Mitapivat (AG-348)

  Update: 25 November 2019 No update available.   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. ...

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Concluded/Terminated Studies-Trials

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 ...

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Sickle Cell Disease

CRISPR gene editing (SCD)   Update: 25 November 2019 CRISPR Therapeutics and Vertex Pharmaceuticals announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001. 1 patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this ...

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Apotransferrin

  Update: 25 November 2019 No update available.   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 ...

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Ferroportin inhibitors

VIT-2763   Update: 25 November 2019 No update available.   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, ...

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SLN-124

  Update: 25 November 2019 No update available.   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 ...

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Denosumab

  Update: 25 November 2019 No update available.   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 ...

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Luspatercept

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 25 November 2019 The U.S. Food and Drug Administration (FDA) has approved REBLOZYL®(luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. The first and only FDA-approved erythroid maturation agent, ...

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Gene Therapy

ZYNTEGLO (ex-Lentiglobin) gene therapy for patients with TDT   Update: 25 November 2019 Manufacturing specifications have been refined in order to aim for the first ‘commercial’ patient to undergo treatment in early 2020. Designation of qualified centres in the EU, signing paperwork and training is underway and will be announced ...

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