05-01-2012: Amsterdam Molecular Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory approval. Orphan designation for AMT’s hemophilia program in the European Union was granted in November 2011.
AMT’s hemophilia B program, which consists of an adeno-associated viral (AAV) vector containing the human factor IX gene, is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital (Memphis, USA) and University College London (UK). Promising data from an initial 6 patients, recently published in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365), shows that gene therapy administration resulted in a reduced need for protein replacement treatment, the standard care for hemophilia patients. AMT is preparing for additional clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.
Amsterdam Molecular Therapeutics (AMT) Holding N.V. in liquidation (in liquidatie), announced that following the approval of the corporate restructuring and financing transaction by the Extraordinary General Meeting ("EGM") of shareholders that was held on 30 March 2012, completion of the d ... more
Amsterdam Molecular Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory appr ... more
Amsterdam Molecular Therapeutics announced that the Company has raised € 2.5 million in new equity by means of a private placement to three of its existing shareholders: Forbion Capital Partners, Gilde Healthcare Partners and Advent Venture Partners. Under the terms of the transaction, AMT ... more
Founded in 1998 on the initiative of researchers from the Academic Medical Center (AMC) in Amsterdam, AMT has become a leader in the field of human gene therapy. A pioneer in the development of gene therapy AMT has used its scientific know-how and expertise to build up a strong research and ... more
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