Navigation Links
Genetix Pharmaceuticals' Adrenoleukodystrophy (ALD) Product Recognized As One of Science Magazine's Top 10 Scientific Breakthroughs of 2009
Date:1/12/2010

ALD Patients Featured in Science Article Now Living Healthy and Normal Lives Due to Groundbreaking Gene Therapy

Cambridge, MA (Vocus) January 12, 2010 -- Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today that Science Magazine has honored the ALD gene therapy research recently published by its collaboratiors at National Institute of Health and Medical Research (INSERM), placing it on their list of ten “Scientific Breakthroughs of 2009.” Genetix is developing this gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of INSERM, and planning to file an IND to expand the development in the US, as well as to expand the ongoing trial in France. The results spotlighted in the Science article show that this innovative therapy has been used to stabilize an otherwise devastating disease in two boys called adrenoleukodystrophy (ALD), or Lorenzo’s Oil disease, in addition to providing a safer alternative to previous therapies.

Adrenoleukodystrophy (ALD) affects 1 in 17,000 males, resulting in severe cognitive and neurologic deficits and eventually leading to death if untreated. It currently is treated with allogeneic bone marrow transplantation, often leading to further complications for the afflicted. In 50% of the patients who survive, graft-versus-host disease or delayed hematological reconstitution delay the time at which disease is stabilized, allowing the disease to have progressed further before stabilization.

Alfred Slanetz, Chief Executive Officer of Genetix, commented, “We are excited and honored that Science Magazine has recognized the ALD gene therapy product. We look forward to further developing this cutting-edge gene therapy in our own clinical trials, and bringing along a therapy that may be curative to this devastating and often terminal disease. I too would like to acknowledge the pioneering and steadfast work of Patrick Aubourg and Doctor Nathalie Cartier, with whom we will continue to work closely as our research and development progresses.”

To view the Science article that recognizes the work of Genetix and INSERM, please click on the following link. http://www.sciencemag.org/cgi/content/full/326/5960/1600

About Genetix Pharmaceuticals
Genetix is the leading global gene therapy company developing curative therapies for genetic diseases. The company’s proprietary platform treats the cause of genetic diseases by placing a healthy gene into the patient's extracted bone marrow stem cells, and transplanting these corrected stem cells back into the patient. Genetix's lead clinical product is Lenti-D™ for Adrenoleukodystrophy (ALD), also known as Lorenzo's Oil disease, which is now in clinical development. The results of the ongoing ALD trial, based upon work conducted by the National Institute of Health and Medical Research (INSERM), were named to Science Magazine's "Scientific Breakthroughs of 2009." Genetix has in-licensed the technology from INSERM, and plans to file for pivotal studies in 2010 in both the U.S. and Europe. In addition to ALD, Genetix is conducting Phase I/II trials with its LentiGlobin™ product in Thalassemia/Sickle Cell Disease, the two most prevalent human genetic disorders. To date, all patients receiving the full therapy with both products have been cured and are healthy. The company’s proprietary stem cell processing, GMP manufacturing and gene therapy vector technologies are also applicable to treating other genetic diseases moving forward. Genetix is privately financed by an experienced syndicate of healthcare investors, including TVM Capital, Forbion Capital Partners and Easton Capital. For more information, please visit http://www.genetixpharm.com/.

###

Read the full story at http://www.prweb.com/releases/2010/01/prweb3427274.htm.


'/>"/>
Source: PRWeb
Copyright©2010 Vocus, Inc.
All rights reserved  

Related medicine news :

1. Imagenetix Enters into Business Relationship with MonaVie
2. Imagenetix, Inc. Reports Third Quarter and Nine Month Results
3. Imagenetix, Inc. Reports Year End Results
4. CetylMax(R) and Flexadren(R) Win Federal Lawsuit - Court Rules Against Imagenetix
5. Imagenetix (IAGX) Obtains Apology
6. 2 children suffering from adrenoleukodystrophy saved thanks to the ELA Association
7. The ScottCare Corporation Completes Acquisition of Rozinn Electronics, a Leading Manufacturer of Cardiac Diagnostic Products
8. Physician Skin Care Specialist Says Proposed New Rules for Sunscreen Products Will Better Protect the Public
9. Watson Receives First FDA Approval for Manufacturing Product at Its Goa, India Facility
10. FDA Seeks to Regulate Complementary and Alternative Medicine; Products Such as Vegetable Juice Could Be Restricted for Medical Use
11. End-to-End HR Performance Benchmarks: Metrics That Will Help Turn the HR Cost Center into a Productivity Driver
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Genetix Pharmaceuticals' Adrenoleukodystrophy (ALD) Product Recognized As One of Science Magazine's Top 10 Scientific Breakthroughs of 2009
(Date:6/24/2016)... CA (PRWEB) , ... June 24, 2016 , ... A ... procedures that most people are unfamiliar with. The article goes on to state that ... but also many of these less common operations such as calf and cheek reduction. ...
(Date:6/24/2016)... ... 2016 , ... June 19, 2016 is World Sickle Cell Observance Day. In ... benefits of holistic treatments, Serenity Recovery Center of Marne, Michigan, has issued ... Sickle Cell Disease (SCD) is a disorder of the red blood cells, which can ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Pulmonary Hypertension ... that it will receive two significant new grants to support its work to ... its 25th anniversary by recognizing patients, medical professionals and scientists for their work ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... ... now offering micro-osteoperforation for accelerated orthodontic treatment. Dr. Cheng has extensive experience with ... Damon brackets , AcceleDent, and accelerated osteogenic orthodontics. , Micro-osteoperforation is a ...
(Date:6/24/2016)... , ... June 24, 2016 , ... Topical BioMedics, Inc, makers of Topricin and MyPainAway ... call for a minimum wage raise to $12 an hour by 2020 and then adjusting ... restore the lost value of the minimum wage, assure the wage floor does not erode ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... , June 23, 2016 Research ... MEMS Devices Medical Market Analysis 2016 - Forecast to 2022" ... The report contains up to date financial data ... analysis. Assessment of major trends with potential impact on the ... analysis of market segmentation which comprises of sub markets, regional ...
(Date:6/23/2016)... MOUNTAIN VIEW, Calif. , June 23, 2016 ... 9:00 a.m. CST on Thursday, July 7, 2016 , , ... kayla.belcher@frost.com ) , , , , EXPERT PANELISTS:  ... Sciences, Nitin Naik; Senior Industry Analyst, Christi Bird; Senior Industry Analyst, ... The global pharmaceutical industry is witnessing ...
(Date:6/23/2016)... , June 23, 2016  Guerbet announced today that ... Supplier Horizon Award . One of ... was recognized for its support of Premier members through ... clinical excellence, and commitment to lower costs. ... this recognition of our outstanding customer service from Premier," ...
Breaking Medicine Technology: