Navigation Links
Amsterdam Molecular Therapeutics Receives Orphan Designation in the European Union for Hemophilia B Gene Therapy
Date:11/15/2011

AMSTERDAM, November 16, 2011 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

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 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.

"Orphan designation is an important milestone for our hemophilia B gene therapy program and will provide additional support to our negotiations as we seek potential licensing partners," said Jörn Aldag, CEO of AMT. "A successful gene therapy for hemophilia could dramatically change not only the lives of patients but also the current hemophilia market that is dominated by protein replacement therapies."  

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 medicine technology :

1. Amsterdam Molecular Therapeutics Glybera(R) Significantly Reduces the Risk of Pancreatitis in LPLD Patients
2. Amsterdam Molecular Therapeutics Receives Opinion on Glybera(R) Marketing Authorisation Application
3. Pharma and Food Thought Leaders Set to Converge in Amsterdam Next April 2011
4. Amsterdam Molecular Therapeutics Reports Promising Data From Cholesterol Lowering Gene Therapy Study
5. Pieris Announces Results and Successful Completion of its PRS-050 Anticalin Phase I Trial at AACR-EORTC-NCI Molecular Targets and Cancer Therapeutics Conference
6. GenturaDx to Host Poster Presentation at Association for Molecular Pathology 2011 Annual Meeting
7. TetraLogic Pharmaceuticals Announces Presentation of Data on Smac Mimetic TL32711 at AACR-NCI-EORTC International Conference: Molecular Targets and Cancer Therapeutics
8. Multiple Programs from Merrimack Pharmaceuticals Pipeline to be Presented at the 2011 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
9. Molecular Devices Launches the MicroMax Low Volume Microplate, the Highest Throughput Low Volume Solution Available
10. Pharmacodynamic Data from Syndax Pharmaceuticals ENCORE 301 Study in Advanced Breast Cancer to be Highlighted at the AACR Molecular Targets and Cancer Therapeutics Conference 2011
11. Telik Announces Ezatiostat (Telintra®) Preclinical Presentation at the 2011 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... , June 24, 2016  Collagen Matrix, ... design, development and manufacturing of collagen and mineral ... announced today that Bill Messer has ... Marketing to further leverage the growing portfolio of ... devices. Bill joins the Collagen Matrix ...
(Date:6/24/2016)... -- The Academy of Managed Care Pharmacy (AMCP) today ... allow biopharmaceutical companies to more easily share health care ... coverage decisions, a move that addresses the growing need ... The recommendations address restrictions in the sharing of product ... a prohibition that hinders decision makers from accessing HCEI ...
(Date:6/24/2016)... 2016   Pulmatrix, Inc ., (NASDAQ: ... drugs, announced today that it was added to the ... its comprehensive set of U.S. and global equity indexes ... important milestone for Pulmatrix," said Chief Executive Officer ... our progress in developing drugs for crucial unmet medical ...
Breaking Medicine Technology:
(Date:6/26/2016)... ... June 26, 2016 , ... PawPaws brand pet supplements ... that was developed to enhance the health of felines. The formula is all-natural and ... main herbs in the PawPaws Cat Kidney Support Supplement Soft Chews are ...
(Date:6/25/2016)... ... , ... Austin residents seeking Mohs surgery services, can now turn to Dr. ... Russell Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization to include ... in Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction of Glenn ...
(Date:6/25/2016)... CA (PRWEB) , ... June 25, 2016 , ... As ... with Magna Cum Laude and his M.D from the David Geffen School of Medicine ... and returned to Los Angeles to complete his fellowship in hematology/oncology at the UCLA-Olive ...
(Date:6/24/2016)... ... June 24, 2016 , ... A recent article published June 14 ... The article goes on to state that individuals are now more comfortable seeking to ... operations such as calf and cheek reduction. The Los Angeles area medical group, Beverly ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Pulmonary Hypertension Association (PHA) ... will receive two significant new grants to support its work to advance research ... anniversary by recognizing patients, medical professionals and scientists for their work in fighting ...
Breaking Medicine News(10 mins):