EGT - company working on cures for Orphan Diseases

Hi all,

My wife and I came across a website for Errant Gene Therapeutics, LLC (based in Chicago) that works towards development of treatments for orphan diseases. One of their areas of research is gene therapy for thalassemia and one of their lead product candidates is Thalagen, that targets Thalassemia.

The relevant webpage for the company is:

http://www.errantgene.com/About_Errant_Gene-Company-Profile.shtml

============================
Errant Gene Therapeutics, LLC™ was established in October 2003 by Patrick Girondi and Norbert L. Wiech, Ph.D. to develop treatments for life-threatening diseases, in particular those recognized as rare disorders or orphan diseases.


EGT’s primary strategy is to address the unmet medical needs of patients with orphan diseases through the development of its technology and treatments. In addition, the company maintains a constant search for new therapeutic advancements that have the potential to be developed into future treatments. The company's strong intellectual property position is the result of world-wide exclusive licensing agreements from prominent academic laboratories and the acquisition of proprietary technology.

The EGT lead product candidates, Thalagen and CG 1521, target the treatment of the blood disorder thalassemia and refractory prostate cancer, respectively. These products are based on pharmacological technology which corrects the errant gene expression causing each disorder. EGT is currently developing two technologies which can be rapidly introduced into human clinical trials, studied and made available to patients. Thalagen is a treatment for thalassemia based on the technology of gene therapy; CG 1521 is an inhibitor of histone deacetylase and represents a new treatment paradigm for therapy resistant prostate cancer.


Additional product candidates in development target pulmonary and neurodegenerative disorders.
===========================

Some more info on their product pipelines and research are available at:

http://www.errantgene.com/Product_Pipeline-Overview.shtml
http://www.errantgene.com/Product_Pipeline-Thalagen.shtml

Some information on Patrick Girondi is available on his webpage at:

http://www.patgirondi.com/For_my_Friends/about.php

What do you guys think about this? Has anyone else heard about them - maybe CAF or any other thalassemia research organization in the US?

Regards,

Bostonian

Hello Bostonian

I emailed them last July and they said that Thalagene is not yet available and that they are starting the human clinical trails and they adviced me to keep checking their site for any further develpment.

Hope this helps
Manal

Hi Bostonian and Manal,

All I know is, I am keeping my fingers triple crossed, and I am very excited about the potential of a cure SOON!!!
  :wootwoot

Hello Team,

Here is an update on the Approval for RAC in June 2007

From:- http://www.errantgene.com/Product_Pipeline-Thalagen.shtml

EGT has secured approval from the The Recombinant DNA Advisory Committee (RAC) to proceed with gene therapy clinical trials for Thalagen™.

Hope the trials starts soon and we get to a cure in the next few years.

Very Interesting, The link from their website shows they are begining phase 1 soon.

http://www.errantgene.com/Product_Pipeline-Overview.shtml

Regards




 

This is really exciting!

I hope that they reach the marketing phase soon.

I just can't wait!

    
I am so happy about this breakthrough. I really hope that the trials would go smoothly and will not be facing any difficulties. After a lifelong journey of being a thalassaemic, i am hoping so much that somebody will find a cure for Thalassaemia. I don't want to get too much excited so soon because i'm afraid it would not work out perfectly. I just don't want the bad news   

A very interesting note about Patrick Girondi, founder of Errant Gene Therapeutics, is that his own son has thalassemia. He has a very personal interest in thalagen research. I wish them the best of luck with their trials.

In our own ongoing poll at http://www.thalassemiapatientsandfriends.com/index.php?topic=144.0 gene research is the highest priority according to those who have voted. After speaking with Dr Sadelain at the Dubai TIF conference in 2006, I came away with the impression that gene therapy will provide a cure for thalassemia. I do not know how long it will take from beginning trials to eventual approval, and it will most likely be years, but for those who are daring, once these trials begin, there will be a need for patients to participate. This may be the only way to attempt this cure for many years to come.

With this in mind, how many patients here would be willing to take part in such a potentially great trial which may lead to them being cured, but with unknown risks, but also pioneering what might be a cure for ALL thals? Would you be willing to participate?

Count me in!

We all have to die one day so why not die trying? If everything goes well maybe they can actually cure the test subjects with no side effects! If not then at least they would know what went wrong and take it into account for the future.

It's a sacrifice that one has to make for the future generations just like the sacrifices that our forefathers made for us in many aspects.

Right said Sajid  . I am also willing to give it a try. I know that it have it own risks and all...but as an older patients, i have been on trials wether i know of it or not.

With Desferals and transfusions in the early 1980's that too have gone through a lot of changes during these years. And that is a trial in itself. Even exjade is a trial on it's own even if it is being used widely in some countries. So i am willing to take part in the Gene therapy. I want a cure of Thalassaemia to come quickly.

It's also a good thing that Patrick Girondi's son have Thalassaemia. At first, I am wondering how Thalassaemia get's the spotlight on being researched. Now i know  :smile

You will find that many of the leaders of the thal organizations around the world have also been affected by thalassemia in some way, often having a child with thal. I found it interesting that in the Maldives, much more attention was paid to thal when the head of the government had a thal grand child. In spite of the enormous problem in Maldives, thal care is a relatively new development there and is why the oldest Maldivian patients (Bassam and Niuma, two dear friends) are only 27. (Happy belated birthday Niuma!  ).