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Ferroportin inhibitors

  VIT-2763  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 shown positive results with a consistent lower serum ...

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Voxelotor

  Update: 01 February 2019 The preliminary results from phase IIa HOPE trials for the safety and efficacy of voxelotor in SCD were presented at the 2018 ASH Annual Meeting. Results reflected the findings of two randomized, multi-centred trials with 154 patients participating – one with adults and adolescents over ...

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Sevuparin

  Update: 01 February 2019 Sevuparin is a new compound based on the commonly used blood thinner, heparin. It is able to restore blood flow and prevent obstruction of small blood vessels due to its anti-adhesive and anti-inflammatory characteristics. Received Orphan Drug Designation by FDA and EMA. A multi-centre, phase ...

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SLN-124

  Update: 01 February 2019 Silence Therapeutics is working on the development of therapeutic RNA molecules that use the body’s natural mechanism of RNAi to stop (or silence) targeted / specific genes in the genome from producing disease-causing proteins (i.e. gene silencing). RNAi is the natural regulation of gene expression ...

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Denosumab

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 -2.5 and -4 in at least one of three examined areas — lumbar spine, femoral neck or wrist bone, was conducted in Greece.  The study ...

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Luspatercept

‘BELIEVE’ Study in Adults with TDT β-thalassaemia   Update: 02 October 2018 Luspatercept is an erythroid maturation agent (EMA) that is believed to regulate late-stage red blood cell maturation. Results were announced from the Phase 3, randomized, double-blind, multi-centre ‘BELIEVE’ clinical study for the evaluation of efficacy and safety of ...

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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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Gene Therapy

LentiGlobin gene therapy for patients with TDT   Update: 01 February 2019 The developing company has proposed an instalments model for the payment of gene therapy. This essentially entails a 20% payment up front before the gene therapy is applied and the remaining 80% to be paid if the treatment ...

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Gene Editing

CRISPR gene editing   Update: 01 February 2019 The FDA has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). The Fast Track Program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical ...

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Hepcidin

Hepcidin trials – TRANSCEND   Update: 01 February 2019 Hepcidin is a hormone that performs various functions related to iron absorption and distribution in the body.  Thalassaemia patients have low hepcidin levels due to iron overload. Abnormally low hepcidin levels caused by genetic mutations or secondary pathology can be replaced ...

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