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Clinical trial updates

Rapamycin

  Update: 31 March 2020 No update available.   Update: 31 January 2020 No update available   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, ...

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Mitapivat (AG-348)

Mitapivat (AG-348)   Update: 31 March 2020 No update available.   Update: 31 January 2020 The clinical proof-of-concept has been established based on a preliminary analysis of the Phase 2 trial of Mitapivat (AG-348) in patients with non-transfusion-dependent thalassaemia announced at the 61st Annual Meeting of the American Society of ...

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Sickle Cell Disease

Imatinib   Update: 14 May 2020 A phase I clinical trial in the US aims to investigate whether imatinib (a SYK tyrosine kinase inhibitor) can reduce the expression of SCD symptoms (e.g. RBC deformability, adhesion / stickiness etc) and thus painful crises in patients with SCD. The hypothesis focuses on ...

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Apotransferrin

  Update: 31 March 2020 No update available.   Update: 31 January 2020 No update available   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 ...

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Ferroportin inhibitors

VIT-2763   Update: 31 March 2020 No update available.   Update: 31 January 2020 No update available.   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 ...

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SLN-124

SLN-124   Update: 31 March 2020 In a corporate presentation, Silence Therapeutics has identified its 2019 accomplishments as: the initiation of a multi-centred, single blind, randomized Phase Ib clinical trial for assessing safety, tolerability, pharmacodynamics and pharmacokinetics of SLN-124 in patients with NTD β-thalassaemia or HbE/ β-thalassaemia receiving orphan drug ...

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Denosumab

  Update: 31 March 2020 No update available.   Update: 31 January 2020 No update available   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 ...

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Reblozyl

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 31 March 2020 No update available.   Update: 31 January 2020 Data evaluating Reblozyl® (ex-Luspatercept-aamt) were presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida. 1% of patients (101/224 patients receiving Luspatercept-aamt in the phase 3 ...

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Gene Therapy

ZYNTEGLO (ex-Lentiglobin) for patients with TDT   Update: 31 March 2020 BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – Discussions with the FDA are underway so the BLA submission is completed in the second half of 2020. However these timelines are likely to be impacted by the COVID-19 situation, moving them to ...

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Gene Editing

CRISPR gene editing   Update: 31 March 2020 No update available.   Update: 31 January 2020 No update available   Update: 25 November 2019 CRISPR Therapeutics and Vertex Pharmaceuticals announced positive, interim data from the first two patients with severe haemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001. 1 ...

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