Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

被引：8

作者：

Pasi, K. John ^{[1
]}

Georgiev, Pencho ^{[2
]}

Mant, Tim ^{[3
]}

Lissitchkov, Toshko ^{[4
]}

Creagh, Michael Desmond ^{[5
]}

Bevan, David ^{[6
]}

Austin, Steve ^{[7
]}

Hay, Charles R. ^{[8
]}

Hegemann, Inga ^{[9
]}

Kazmi, Rashid ^{[10
]}

Chowdary, Pratima ^{[11
,12
]}

Ragni, Margaret V. ^{[13
,14
]}

Soh, Chang-Heok ^{[15
]}

Akinc, Akin ^{[15
]}

Partisano, Angela M. ^{[15
]}

Sorenson, Benny ^{[15
]}

Rangarajan, Savita ^{[16
]}

机构：

[1] Barts & London Queen Marys Sch Med & Dent, Royal London Haemophilia Ctr, London, England

[2] Univ Multiprofile Hosp Act Treatment Sveti Georgi, Plovdiv, Bulgaria

[3] Quintiles Drug Res Unit, London, England

[4] SHAT Joan Pavel, Dept Coagulat Disorders & Anemia, Sofia, Bulgaria

[5] Royal Cornwall Hosp NHS Trust, Truro, England

[6] Guys & St Thomas Hosp NHS Trust, London, England

[7] St Georges Univ Hosp NHS Fdn Trust, St Georges Haemophilia Ctr, London, England

[8] Manchester Royal Infirm, Manchester, Lancs, England

[9] Univ Hosp, Zurich, Switzerland

[10] Univ Hosp Southampton NHS Fdn Trust, Southampton, Hants, England

[11] Royal Free Hosp, KD Haemophilia Ctr, London, England

[12] Royal Free Hosp, Thrombosis Unit, London, England

[13] Univ Pittsburgh, Div Hematol Oncol, Dept Med, Med Ctr, Pittsburgh, PA USA

[14] Univ Pittsburgh, Hemophilia Ctr Western PA, Med Ctr, Pittsburgh, PA USA

[15] Alnylam Pharmaceut, Cambridge, MA USA

[16] Guys & St Thomas NHS Fdn Trust, Ctr Haemostasis Thrombosis, London, England

来源：

BLOOD | 2016年 / 128卷 / 22期

关键词：

D O I：

10.1182/blood.V128.22.1397.1397

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

1397

引用

页数：6

共 50 条

[1] Phase 1 and Phase 1/2 Extension Study of Fitusiran, an Investigational Rnai Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results in Patients With Inhibitors
Pasi, K. J.
Georgiev, P.
Mant, T.
Creagh, M. D.
Lissitchkov, T.
Bevan, D.
Austin, S.
Hay, C. R.
Hegemann, I.
Kazmi, R.
Chowdary, P.
Ragni, M. V.
Soh, C. -H.
Akinc, A.
Monpara, A.
Madigan, K.
Rangarajan, S.
HAEMOPHILIA, 2017, 23 : 88 - 89
[2] Phase 1 and Phase 1/2 Extension Study of Fitusiran, an Investigational Rnai Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results in Patients Without Inhibitors
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Georgiev, P.
Mant, T.
Creagh, M. D.
Lissitchkov, T.
Bevan, D.
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Hay, C. R.
Hegemann, I.
Kazmi, R.
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[3] Management of Bleed Events in the Phase 1 Study of Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia in Patients with and Without Inhibitors
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[4] Perioperative management in patients with hemophilia receiving fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia
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[5] Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia
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[6] Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort
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[7] A subcutaneously administered investigational RNAi therapeutic (ALN-AT3) targeting antithrombin for treatment of hemophilia: interim phase 1 study results in patients with hemophilia A or B
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[8] A Subcutaneously Administered Investigational RNAi Therapeutic, Fitusiran (ALN-AT3), Targeting Antithrombin for Treatment of Hemophilia: Interim Results in Patients with Hemophilia A or B
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[9] Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)
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[10] Thrombin generation response with the addition of bypassing agents in plasma of patients treated with fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia
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