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

3

GOOD

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:11/30/2009)...30 BioMarin Pharmaceutic...Food and Drug Administration (FDA) has granted orp...DAP), amifampridine phosphate, for the rare autoim...MS). 3,4-DAP has previously received orphan drug ...e Committee for Medicinal Products for Human Use o...
(Date:11/30/2009)...GEN, Nov. 30 Exiqon A/S (NASDAQ OM...ard Medical School ICCB-Longwood Screening Facilit...ning Center of Excellence. ICCB-L supports invest...hospitals and institutions, for high-throughput sc..., MicroRNAs are a novel class of small, non-coding...
(Date:11/30/2009)..., AMSTERDAM, November 30 /PRNewswire-FirstC... scientific, medical and technical products and se...e, its comprehensive biomedical,database which hol...7,000,active, peer reviewed journals. , E...t of new biomedical,and pharmaceutical insights. N...
(Date:11/27/2009)...Nov. 27 Cromoz Inc., located in Re...carbon nanotube-based cancer drug delivery system ...tubes, which have functional groups on the walls t...developed in partnership with the Indian Institute...serves as a drug delivery vehicle with the ability...
Breaking Biology Technology:FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS 2FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS 3FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS 4Exiqon Recognizes Harvard Medical School Core Facility as MicroRNA Screening Center of Excellence 2Exiqon Recognizes Harvard Medical School Core Facility as MicroRNA Screening Center of Excellence 3Elsevier is Re-Launching "Embase" to be a Powerful Resource Providing Deep Insights and Answers to Biomedical Researchers 2Elsevier is Re-Launching "Embase" to be a Powerful Resource Providing Deep Insights and Answers to Biomedical Researchers 3North Carolina Based Cromoz Inc. Launches Carbon Nanotechnology for Target Drug Delivery System in Cancer Treatment in Hyderabad, India 2
...e next installment covering the enforcement tactic...week. , , You or your company may have no paten...cerned with patent matters. However, there is a gr...tents to be a main or significant source of revenu... to be concerned with patent matters. , ,In the pa...
...ital Forum has issued an open call for companies ...re venture capitalists, corporate and private inve...thering April 5-6. , ,Dec. 16 is the early applica...n deadline to be considered for participation in t...nal headquarters in St. Louis. , ,Selection of pre...
...z about how gaming is going to transform corporate...em Advanced Distributed Learning Co-Lab are doin...knowledge, learning and industry. , ,Guess what? ... reroute the focus of innovation and development f... great time of year to get a feel for the scope an...
Other Biology Technology:Patent enforcement: What you don't know might hurt you 2Patent enforcement: What you don't know might hurt you 3Patent enforcement: What you don't know might hurt you 4Just in time for Christmas: Retail IT. 2Just in time for Christmas: Retail IT. 3
(Date:11/30/2009)...d to monitor the commitments that will be made by ...forestation rates, according to research at the Un...s and research groups have failed to make full use...,s surface collected each week to monitor tropical...Grainger in the Proceedings of the National Acade...
(Date:11/30/2009)...state of Antarctica,s climate and its relationship...ek (Tuesday 1 December) by the Scientific Committe...rctic Climate Change and the Environment presents...ifies areas for future scientific research, and ad...ve about Antarctic melting, sea-level rise and bio...
(Date:11/30/2009)...ert at the holiday table are even less of a help t...g to a new research report recently appearing onli...), a diet that is high in fat and in sugar actuall...es to store too much fat. This means these foods h...lt task of converting high-fat and high-sugar food...
Breaking Biology News(10 mins):World forest observatory needed to monitor vital role of forests in climate deal 2First comprehensive review of the state of Antarctica's climate 2Happy holidays from the groaning board; Western diets turn on fat genes 2Radiation Plus Chemo a Good Option in Lung Cancer 52797 1Radiation Plus Chemo a Good Option in Lung Cancer 52797 2Grant for brain tumor and deafness research 52795 1Grant for brain tumor and deafness research 52795 2Genetic testing may be valuable in treating colorectal cancer 52793 1Genetic testing may be valuable in treating colorectal cancer 52793 2
...HO, N.Y., June 5 bioMETRX Inc., (OTC,Bulletin Boa...sumer-based,finger-activated products, today annou...ric Solutions, LLC, to co-develop a new generation... technology to consumers at,an accelerated rate, g..."Biometric Solutions, LLC has had successful engin...
...research team has uncovered the likely target of n...ide a clearer picture of how this vitamin helps ma...d and thus lower the risk of heart disease. , Whi...anism of how it works has been mysterious, althoug...ease HDL production. Recent work had uncovered tha...
...This release is available in German . , Leipzig....mental Research have developed two new methods, in...f European Otters (Lutra lutra) and their effects ...eeded for the first time in gathering more accurat...nd region of the Oberlausitz Biosphere Reserve. Ge...
Other Biology News:bioMETRX, Inc. Partners with Biometric Solutions, LLC to Deliver Next Generation Finger Activated Technology 2bioMETRX, Inc. Partners with Biometric Solutions, LLC to Deliver Next Generation Finger Activated Technology 3Otters reveal their identity 2Otters reveal their identity 3Otters reveal their identity 4
... DuoSet ELISA Development kit contains the basic c... ELISAs to measure natural and recombinant mouse D...es and serum. Each kit contains sufficient materia...plates, provided that the following conditions are...
pCDF-1 Expression System plus Competent Cells from Novagen
Chicken Anti-Human Uridine Phosphorylase 1 Polyclonal Antibody, Unconjugated from Abcam
RayBio Mouse AngiogenesisAntibody Array I & 1.1 Service from Raybiotech, Inc.
Biology Products: