Publications

  Update: 29 March 2019 Developed for sickle cell disease, IMR-687 is a highly potent, selective inhibitor of phosphodiesterase-9 (PDE9i) in blood cells. It targets the same biochemical pathway as hydroxyurea, a chemotherapeutic agent, but without its safety issues. IMR-687 has been shown in cell and animal models to increase ...

  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 ...

  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 ...

  Update: 29 March 2019 The first of three cohorts has been successfully dosed in a phase I study to assess the safety of subcutaneous administration of Sevuparin for the treatment of SCD. This double-blind, placebo-controlled Phase I study, complements the currently ongoing Phase II study which is examining the ...

  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 first patient expected to enter the study in ...

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 ...

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia Update: 29 March 2019 Regulatory updates have been announced indicating that Luspatercept Biologics License Application (BLA) will be submitted in April 2019. A BLA  is submitted after an investigational new drug has been approved and constitutes a request for permission to introduce, or deliver ...

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 ...

LentiGlobin gene therapy for patients with TDT   Update: 29 March 2019 The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines  Agency (EMA) adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO™ (autologous CD34+ cells encoding β A-T87Q-globin gene), a gene therapy for patients 12 years and older with ...

CRISPR gene editing   Update: 29 March 2019 The Phase 1/2 Study in Beta Thalassaemia 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 transfusion-dependent thalassaemia (TDT), with non-β0/β0genotype subtypes. Two patients will be initially ...