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:4/24/2017)... ... 2017 , ... The John P. McGovern Museum of Health and Medical Science ... their cognitive health, and share results with their physicians. Members and guests can sign ... website. , BrainCheck founder, Dr. David Eagleman, formerly at Baylor College of Medicine and ...
(Date:4/24/2017)... ... April 24, 2017 , ... ... seeking public support to bring their novel lifesaving device for the everyday use ... with medical-grade sensors, specially designed to read a child’s vital signs, and detect ...
(Date:4/24/2017)... ... April 24, 2017 , ... Salus Telehealth, Inc. ... company’s VideoMedicine mobile platform has launched Quick Care, a new service offering on-demand ... Care provides patients with the option to request and begin a consultation with ...
(Date:4/24/2017)... (PRWEB) , ... April 24, 2017 , ... ... (CDC), rising prescription opioid overdose deaths now claim the lives of 62 Americans ... civil case filings against drug manufacturers, distributors, pharmacies and prescribers by more than ...
(Date:4/24/2017)... Tx (PRWEB) , ... April 24, 2017 , ... ... Lief Therapeutics designed to recognize elevated anxiety levels in people with addiction who ... the Lief Smart Patch, a biosensing wearable device that monitors heart and breath ...
Breaking Medicine News(10 mins):
(Date:4/20/2017)... , April 20, 2017  Vivify Health, the pioneer ... has been awarded a very significant patent for the ... to continual care via digital health.  This landmark patent ... property and further secures Vivify,s position as the leader ... 2009, was the first company to apply consumer mobile ...
(Date:4/20/2017)... April 20, 2017 Research and Markets has ... Innovation Driven by Rapidly Expanding Injectables Market and Increasing Usage of ... ... delivery technologies will rise from USD 20 Billion in 2015 to ... Drug Delivery Technologies - Innovation Driven by Rapidly Expanding Injectables Market ...
(Date:4/20/2017)... Research and Markets has announced the ... Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies ... ... was valued at US$ 7,167.6 Mn in 2015, and is ... a CAGR of 5.6% from 2016 to 2024. ...
Breaking Medicine Technology: