Navigation Links
Amsterdam Molecular Therapeutics Receives US Orphan Designation for Hemophilia B Gene Therapy
Date:1/3/2012

AMSTERDAM, January 4, 2012 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today 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.

"U.S. orphan designation provides additional support for our hemophilia B gene therapy program and supplements the designation in the EU received in November," said Jörn Aldag, CEO of AMT. "The early clinical success seen with the program to date by our partners is very encouraging. We will build on this success in the coming months."  

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry.  AMT was founded in 1998 and is based in Amsterdam. Further information can be found at http://www.amtbiopharma.com.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law. 

 



'/>"/>
SOURCE Amsterdam Molecular Therapeutics (AMT) B.V
Copyright©2010 PR Newswire.
All rights reserved

Related biology technology :

1. Amsterdam Molecular Therapeutics Reports Half Year Results 2008
2. Amsterdam Molecular Therapeutics Reports Full Year Results 2008
3. Amsterdam Molecular Therapeutics Reports Half Year Results 2009
4. The University of Amsterdam Joins the MetaMiner Metabolic Diseases Project
5. Amsterdam Molecular Therapeutics Reports Full Year Results 2009
6. Amsterdam Molecular Therapeutics Reports Half-Year Results 2010
7. Amsterdam Molecular Therapeutics Successfully Lowers Cholesterol In Vivo With Enhanced Novel MicroRNA AAV Gene Therapy
8. Amsterdam Molecular Therapeutics Appoints Dr. Carlos R. Camozzi as Chief Medical Officer
9. Amsterdam Molecular Therapeutics Files Glybera® European Marketing Application for Re-examination
10. Amsterdam Molecular Therapeutics Reports Half-Year Results 2011
11. Amsterdam Molecular Therapeutics Licenses Additional Novel Gene Therapy Vectors from National Institutes of Health
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/27/2016)... 2016  Sequenom, Inc. (NASDAQ: SQNM ), ... through the development of innovative products and services, announced ... United States denied its petition to review ... Sequenom,s U.S. Patent No. 6,258,540 (",540 Patent") are not ... the Supreme Court,s Mayo Collaborative Services v. Prometheus Laboratories ...
(Date:6/27/2016)... Raleigh, NC (PRWEB) , ... June 27, 2016 ... ... a mission to bring innovative medical technologies, services and solutions to the healthcare ... development and implementation of various distribution, manufacturing, sales and marketing strategies that are ...
(Date:6/27/2016)... Ginkgo Bioworks , a leading organism design company ... as one of the World Economic Forum,s Technology ... companies. Ginkgo Bioworks is engineering biology to manufacture ... the nutrition, health and consumer goods sectors. The ... Fortune 500 companies to design microbes for their ...
(Date:6/24/2016)... ... June 24, 2016 , ... While the majority of commercial spectrophotometers and ... and the 6000i models are higher end machines that use the more unconventional z-dimension ... light beam from the bottom of the cuvette holder. , FireflySci has developed ...
Breaking Biology Technology:
(Date:4/26/2016)... and LONDON , April ... part of EdgeVerve Systems, a product subsidiary of ... today announced a partnership to integrate the Onegini ...      (Logo: http://photos.prnewswire.com/prnh/20151104/283829LOGO ) ... their customers enhanced security to access and transact ...
(Date:4/14/2016)... 14, 2016 BioCatch ™, ... today announced the appointment of Eyal Goldwerger ... Goldwerger,s leadership appointment comes at a time ... the deployment of its platform at several of the ... which discerns unique cognitive and physiological factors, is a ...
(Date:3/23/2016)... , March 23, 2016 ... erhöhter Sicherheit Gesichts- und Stimmerkennung mit Passwörtern ... (NASDAQ: MESG ), ein führender ... das Unternehmen mit SpeechPro zusammenarbeitet, um erstmals ... Finanzdienstleistungsbranche, wird die Möglichkeit angeboten, im Rahmen ...
Breaking Biology News(10 mins):