Clinical trial updates

Alpha-thalassaemia major

Alpha-thalassaemia major

    Update: 05 November 2020 Researchers at the University of California – San Francisco (UCSF) are conducting a Phase I clinical trial to test a new strategy for conducting…
IMR-687

IMR-687

    Update: 05 November 2020 The first thalassaemia patient has been dosed in the FORTE clinical trial – a Phase 2b clinical trial that will evaluate the safety and…
Rapamycin

Rapamycin

  Update: 05 November 2020 No update available.   Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No…
Mitapivat (AG-348)

Mitapivat (AG-348)

    Update: 05 November 2020 Interim data will be presented at the 62nd ASH Meeting in December 2020. Sources: https://investor.agios.com/news-releases/news-release-details/agios-present-broad-set-clinical-and-translational-data-0   Update: 25 August 2020 FDA Orphan Drug Designation…
Apotransferrin

Apotransferrin

  Update: 05 November 2020 No update available.   Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No…
Ferroportin inhibitors

Ferroportin inhibitors

VIT-2763   Update: 05 November 2020 No update available.   Update: 25 August 2020 Data presented at the EHA Congress in June indicate that treatment with the oral ferroportin inhibitor…
SLN-124

SLN-124

    Update: 05 November 2020 Initiated dosing in randomised, double-blind, placebo-controlled Phase 1 single ascending dose study of SLN124 in up to 24 healthy volunteers with β-thalassaemia and MDS.…
Denosumab

Denosumab

  Update: 05 November 2020 No update available.   Update: 25 August 2020 No update available.   Update: 31 May 2020 No update available.   Update: 31 March 2020 No…
Reblozyl

Reblozyl

  ‘BELIEVE’ Study in Adults with TDT β-thalassaemia     Update: 05 November 2020 Long term safety and efficacy results on REBLOZYL will be presented at the 62nd ASH Meeting…
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