Navigation Links
AMT Starts Collaboration With St. Jude Children's Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy
Date:7/14/2008

ating with them. "

Amit Nathwani, M.D., Ph.D., who initiated his work on Factor IX while in Nienhuis' lab, has continued to collaborate with St. Jude faculty Andrew Davidoff, M.D., and John Gray, Ph.D., in developing a novel AAV vector that produces therapeutic levels of Factor IX when given intravenously in animal models. This vector has been licensed to AMT.

Repair factor IX production and blood clotting

Hemophilia B is characterized by defective blood clotting due to an absence of functional clotting Factor IX. The gene with the information for the Factor IX protein is mutated in Hemophilia patients, resulting in the production of non-functional protein. AMT uses gene therapy to introduce the functional gene into the patient's cells and thereby restore blood clotting.

AMT recently acquired a license from 'TIGET' San Raffaele Telethon Institute For Gene Therapy, Italy, to use its micro-RNA technology to prevent immune responses against gene therapy for Hemophilia B. AMT will use the TIGET technology to prevent an immune response against the newly formed Factor IX, which the patient's body may recognize as being foreign.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially the ones that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development.

About St Jude Children's Research Hospital

St. Jude Children's Research Hospital is internationally recogn
'/>"/>

SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2008 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related biology technology :

1. Anesiva Launches Needle-Free Zingo(TM) to Reduce Pain from IV Starts and Blood Draws in Children
2. Oncolytics Biotech Inc. Starts Patient Enrolment in U.K. Phase II Clinical Trial Investigating REOLYSIN(R) in Combination with Paclitaxel and Carboplatin
3. Oncolytics Biotech Inc. Starts Patient Enrolment in U.K. Combination REOLYSIN(R)/Cyclophosphamide Trial
4. Synosia Starts Phase II Efficacy Trial For Rufinamide
5. Evotec Starts Phase II in Smoking Cessation with EVT 302
6. Carrington Subsidiary DelSite Starts Making Test Batches of First Bird Flu Vaccine Offering Needle-free Self-Administration
7. Sigma-Aldrich Announces Collaboration With Metahelix Life Sciences
8. Pharmaceutical Institute, in Collaboration With Campbell Alliance, Releases Second Edition of Breakthrough Book on the Inner Workings of the Pharmaceutical Industry
9. SENOMYX ANNOUNCES EXTENSION OF DISCOVERY AND DEVELOPMENT COLLABORATION WITH CADBURY PLC
10. ISMPP Applauds AAMC Clarification on Ghostwriting Versus Transparent Writing Collaboration in Scientific Publications
11. Exelixis and GlaxoSmithKline Agree to Successfully Conclude Six-Year Discovery and Development Collaboration
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/10/2014)... July 10, 2014   Ceres, Inc . (Nasdaq: ... company, announced today financial results for the three months ... its business. Ceres reported that the company ... performance this growing season in Brazil, which concluded in ... company,s sorghum evaluation areas for part of the growing ...
(Date:7/10/2014)... of Adelaide research may help in the fight against ... detect tiny quantities of explosives with the use of ... journal Sensors and Actuators B: Chemical , the ... detect explosives in concentrations as low as 6.3 ppm ... only a few minutes. , "Traditionally explosives detection has ...
(Date:7/10/2014)... A new study published today in the journal ... has found that time of day and sleep deprivation ... could be crucial when looking at the best time ... and heart disease, and for administering medicines effectively. , ... of Cancer Research, London, investigated the links between sleep ...
(Date:7/10/2014)... the fractional quantum Hall effect in bilayer graphene and ... tuned by an electric field. , The fractional ... to thin sheets are exposed to large magnetic fields, ... of individual electrons behave as a single system. However, ... established, many details of this collective behavior remain not ...
Breaking Biology Technology:Ceres Announces Fiscal Third Quarter 2014 Financial Results 2Ceres Announces Fiscal Third Quarter 2014 Financial Results 3Ceres Announces Fiscal Third Quarter 2014 Financial Results 4Ceres Announces Fiscal Third Quarter 2014 Financial Results 5Ceres Announces Fiscal Third Quarter 2014 Financial Results 6Ceres Announces Fiscal Third Quarter 2014 Financial Results 7Ceres Announces Fiscal Third Quarter 2014 Financial Results 8Ceres Announces Fiscal Third Quarter 2014 Financial Results 9Detecting trace amounts of explosives with light 2Time of day crucial to accurately test for diseases, new research finds 2Columbia researchers observe tunable quantum behavior in bilayer graphene 2Columbia researchers observe tunable quantum behavior in bilayer graphene 3
... there's always one person in a crowd who is proud ... me. I'm compulsive about reading the job classifieds. , ,Don't ... the difference between the ubiquitous "excellent communication skills" and the ... quick scan every day, even if it happens to be ...
... a maker of automotive and ... bought Pro-Tel Inc. for an ... a contracting company founded in ... 70 employees. Its repair and ...
... Gas & Electric has released its 2005 Greater ... and updated listings covering 394 organizations. , ,The directory, ... offers an overview of the high-tech firms, professional organizations, ... in Dane County, as well as an array of ...
Cached Biology Technology:Is IT leadership a position or an attitude? 2Is IT leadership a position or an attitude? 32005 Madison-area tech directory includes more companies, resources 22005 Madison-area tech directory includes more companies, resources 3
(Date:7/11/2014)... Salk Institute for Biological Studies, BGI, and other ... and reliability of the existing targeted gene correction ... which could significantly increased gene-correction efficiency in human ... online in Cell Stell Cell provides ... therapy. , The combination of stem cells and ...
(Date:7/11/2014)... July 11, 2014 Researchers have pioneered ... images. The new technology, called Virtual Finger, allows ... structures like neurons and synapses using the flat ... technology makes 3D imaging studies orders of magnitude ... an unprecedented level across many areas of experimental ...
(Date:7/11/2014)... the normal circumstances, astrocytes participate in normal ... and exhibit therapeutic and repairing effects on ... have found that nerve cells differentiated from ... reduced viability, which produces influences on subsequent ... General Hospital, Hebei United University, China demonstrated ...
Breaking Biology News(10 mins):A new genome editing method brings the possibility of gene therapies closer to reality 2Virtual finger enables scientists to navigate and analyze complex 3D images 2
... 19, 2012 /PRNewswire-iReach/ -- Global Information ... of forthcoming conferences and networking events in the ... events provide a platform for researchers and decision ... latest findings and learn about significant developments in ...
... have discovered the unexpected way in which a key cell ... said, offers insight into the processes that take place within ... from spinal cord injury to cancer. The research focused ... cell. Like other cells in the immune system, its job ...
... Va.The Office of Naval Research,s (ONR) biennial 2012 Naval ... Oct. 22-24, officials announced Jan. 19. "The chief ... Navy and Marine Corps, vision for 2025," said Rear ... is to bring the right people together to deliver ...
Cached Biology News:2012 Biotechnology and Pharmaceuticals: Forthcoming Conferences and Exhibitions 22012 Biotechnology and Pharmaceuticals: Forthcoming Conferences and Exhibitions 32012 Biotechnology and Pharmaceuticals: Forthcoming Conferences and Exhibitions 4UCSF team uncovers how immune cells move against invaders 2ONR sets date for 2012 Naval S&T Partnership Conference 2
... and G. Butcher (1998). • The major ... research in the immunology area. There is ... proteins and in their function as antigen-presenting ... with the prospect of tackling biochemical and ...
VACUGENE PUMP 110V, 1 EA. Category: Nucleid Acid Detection Systems....
Rat whole blood (Sprague-Dawley, averaging 7-8 weeks of age, either sex) is collected at the time of slaughter and refrigerated within 6 hours, and processed and frozen within 24 hours. The serum is ...
... mutS and ES1301 mutS are mismatch repair ... these strains prevents repair of the newly ... mutation efficiencies and making them helpful in ... Sites II Mutagenesis Systems. Both ES1301 and ...
Biology Products: