AAV5-hFIX

GENETIC2 trials

Sponsors

CSL Behring

Conditions

Hemophilia B

Phase 1

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

CompletedNCT02396342

CSL BehringHemophilia B

Start: 2015-06-10End: 2021-04-15Updated: 2022-06-27

Unknown Phase

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

CompletedNCT05360706

CSL BehringHemophilia B

Start: 2021-03-18End: 2026-03-26Updated: 2026-05-26

Related Papers

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Blood2024-11-054 citations

Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A

Blood Advances2024-07-183 citations

Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Blood2022-11-152 citations

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirms Stable FIX Expression and Sustained Reductions in Bleeding for up to 5 Years

Hämostaseologie2021-06-01

AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years

Blood2020-11-0517 citations

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Blood2019-11-1327 citations

Clearance of Vector DNA Following Systemic Administration of AAV5-hFIX or AAV5-hFIX Padua in Patients with Severe or Moderate-Severe Hemophilia B

Blood2019-11-133 citations

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Blood2018-11-297 citations

Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Blood2017-12-15295 citations