Navigation Links
Genethon and Children's Hospital Boston Get FDA approval for a Wiskott Aldrich Gene Therapy Trial
Date:7/21/2011

BOSTON, July 21, 2011 /PRNewswire-USNewswire/ -- The U.S. Food & Drug Administration (FDA) approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome (WAS). After its implementation in Paris and London, this trial based on preclinical research performed at Genethon (Evry, France) which also manufactures the GMP gene therapy product, is now going to be launched in Boston. It's one of the first international clinical trials using a gene therapy treatment for a rare disease.  

Earlier this year, Genethon, the not-for-profit biotherapy laboratory funded by the French Muscular Dystrophy Association (AFM) thanks to the donations collected during the French Telethon, and Children's Hospital Boston announced that they have initiated a partnership to conduct a gene therapy clinical trial for Wiskott-Aldrich Syndrome (WAS), a severe immunodeficiency disease leading to death before adulthood. Genethon is sponsoring parallel trials at Great Ormond Street Hospital in London and Hopital Necker-Enfants Malades in Paris and is supplying the vector for all clinical sites. In total, this trial will involve fifteen patients, five for each site, who will be treated by 2013-2014. The sites in London and Paris have already started treating patients under this protocol. Altogether, the WAS gene therapy trials in London, Paris and Boston will constitute a unique multicenter collaboration to accelerate the testing of new advanced therapies for rare conditions.

The Boston trial will be funded by the National Heart, Lung and Blood Institute (NHLBI) through its Gene Therapy Resource Program (GTRP). Principal investigators at Children's are Sung-Yun Pai, MD, Division of Hematology/Oncology, and Luigi Notarangelo, MD, director, Research and Molecular Diagnosis Program on Primary Immunodeficiencies, Division of Immunology. David A. Williams, MD, chief of the Division of Hematology/Oncology and director o
'/>"/>

SOURCE Children's Hospital Boston
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related biology technology :

1. Pradaxa® (dabigatran etexilate mesylate) Capsules Now on Formulary at Nations Top Heart Hospitals
2. Axial Biotech Signs Agreement With Shriners Hospital for Children
3. Texas Childrens Hospital is First Pediatric Hospital in the Nation to Implant Artificial Heart
4. Paoli Hospital Pioneers New Treatment for Uterine Fibroids and Malformations
5. Baylor College of Medicine and Texas Childrens Hospital Neuroscientist Dr. Huda Zoghbi Awarded Gruber Neuroscience Prize
6. Hospital for Special Surgery to Host International Arthritis Summit: Frontiers in Osteoarthritis Research, Prevention and Care
7. St. Lukes Hospital & Health Network Selects CareTech Solutions to Provide Proactive Monitoring of Hospital IT Infrastructure
8. BioNeutral to Premier its EPA-Approved Ygiene® 206 Hospital-Grade Disinfectant at the Annual 2011 APIC Conference, While Community Health of South Florida Begins Product Review
9. SynapDx Corporation Licenses Autism Diagnostic Discoveries From Childrens Hospital Boston
10. Newly Published Study Shows Dramatic Reduction in Hospital Acquired Infections
11. WARFARIN Study Launched at WellStar Health System to Assess Impact of Genetic Testing in Reducing Hospitalizations and Deaths Caused by Warfarin
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/22/2014)... Terry Baughn, a senior lecturer in mechanical engineering at ... Engineer of the Year by the North Texas section ... ASME has more than 130,000 members in 158 countries ... technology to assist the engineering community, benefiting the world. ... Texas Society of Professional Engineers honors banquet. Baughn retired ...
(Date:7/22/2014)... Lake, Wisconsin (PRWEB) July 22, 2014 ... be in attendance at the 2014 NCSL International ... Booth #423 will feature CONDEC pressure instrumentation, ... style weights, and the new, customized sets, Essential ... ago to promote cooperative efforts in solving the ...
(Date:7/22/2014)... SHENZHEN , Kina, 22. juli ... (BGI Tech), et datterselskab af BGI, verdens største ... human whole exome-sekvenseringstjeneste baseret på Complete Genomics brancheførende ... i branchen for at levere patenterede sekvenseringsteknologier med ... typer varianter, blev købt af BGI i 2013. ...
(Date:7/22/2014)... 22, 2014 In chemical and ... increasingly common sight. The rapid rise of TDL ... well- established as a core gas measurement technology, ... made them the gas analyzer technology of choice ... encountered in some processes have limited their application ...
Breaking Biology Technology:UT Dallas professor receives Engineer of the Year award 2Rice Lake Weighing Systems Showcases Metrology Solutions for the 2014 NCSL International Workshop & Symposium 2BGI Tech lancerer Human Whole Exome-sekvenseringstjeneste på Complete Genomics avancerede platform 2METTLER TOLEDO Shows How Tunable Diode Lasers (TDLs) Can Be Installed Where No TDLs Have Gone Before 2
... What one thing, if done extremely well, is a ... organizations? And what, if done poorly, is an enormous ... delegate will have a direct impact on how effectively ... article I will share some delegation tips on how ...
... has launched a new service to help the Wisconsin business ... Corporate Relations and University Communications developed UW Business News ... events and links to publications. It is available online or ... one place ... all the sorts of information that would ...
... Summary and conclusion , ,Are your projects still late? ... less than promised? Unfortunately, the answer is probably yes. , ... neednt be the case. Think about it you learn new ... same mistake twice. Youve gone to class. Youve learned the ...
Cached Biology Technology:Fire up your marketing by delegating 2Wire service to connect UW-Madison to business 2Project Performance: Is your project late? Over budget? 2Project Performance: Is your project late? Over budget? 3
(Date:7/22/2014)... some point, most kids will hear that drinking milk helps make ... But do these messages foster the idea that if something is ... new study in the Journal of Consumer Research , when ... to eat it. , "We predicted that when food is presented ... achieve a goal such as learning how to read or count, ...
(Date:7/22/2014)... LA -- Nancy Buccola, MSN, APRN, PMHCNS-BC, CNE, ... Sciences Center New Orleans School of Nursing, contributed ... of genetic material associated with schizophrenia and also ... and schizophrenia. The study, Biological insights from 108 ... 2014 in Nature , available at http://www.nature.com/nature/journal/vaop/ncurrent/full/nature13595.html., ...
(Date:7/22/2014)... meerkats which sees them prevent their daughters from breeding, ... study. , Research into the desert creatures which live ... helpers shows that the alpha female can flourish when ... shows how this way of life, also found in many ... its sinister side. , Dominant meerkats control breeding within ...
Breaking Biology News(10 mins):LSUHSC contributes to work identifying new DNA regions associated with schizophrenia 2
... intervention taught by teen mentors in Appalachian elementary schools ... lifestyle changes among the younger students learning the curriculum, ... taught the same lessons by adults in a traditional ... The results of the eight-week clinical trial conducted by ...
... few years ago, the idea of 3-D printing a major ... eye North Carolina as a national hub for human organs ... Medical advances in biotechnology seem to be coming faster ... how society should handle ethical, legal and moral considerations. ...
... a school classroom chemistry experiment, in which beautiful plant-like structures ... real-world scientific experiments that aim to shed light on the ... a "battery" that could have kick-started life are the topic ... & Engineering News . C&EN is the weekly newsmagazine of ...
Cached Biology News:Teen mentors inspire healthier choices in younger children 2Teen mentors inspire healthier choices in younger children 3Teen mentors inspire healthier choices in younger children 4'After the Genome' tackles tough questions about medicine, miracles and morality 2'After the Genome' tackles tough questions about medicine, miracles and morality 3
... DH-2000, but comes with a shutter (controlled via ... Hz) The DH-2000 Deuterium Tungsten ... deuterium and tungsten halogen light sources in a ... a powerful, stable output from 215-200 nm. In ...
... in g per ml and is determined from ... of a 1 mg per ml solution. ... agglutinate a 2% suspension of human blood group ... C. Preparation Note: Affinity purified by ...
... MSD Security ChemStation is ... software application for data ... library searching and reporting, ... of data security, integrity, ...
... save space and organize gloves, ... other garb. They are the ... small parts used in labs, ... Open-slot design for easy cleaning. ...
Biology Products: