Meeting on gene therapy November 14 from 11:30 a.m. to 1:30 p.m

thank u so mch andy tht u r always there wth thalasaemia fighting for people like us.............god has given u immense strength and wisdom.............thanx so mch would like to hear soon about what the doc says finally on gene therapy

Yeah andy u r our reall hero , and im very thankfull for ur cooperation with us and thank u ones agine ........

Thank you all for your support. It really means a lot to me. I don't like having to challenge research but...sometimes things don't add up.

from Pat:

Dear family members,
 
Some good news that I wanted to share with you. Continue to send warm wishes and prayers. We need them more than ever... I love you all.
 
pat g
 
Errant Gene Therapeutics Awarded $245,000 Under U.S. Qualified Therapeutic Discovery Project Program
Chicago, IL. - November 2, 2010


Errant Gene Therapeutics, LLC (EGT), a biopharmaceutical company headquartered in Chicago , Illinois , announced today that it has been awarded a $245,000 grant under the Qualifying Therapeutic Discovery Project (QTDP) program.

The QTDP, created by U.S. Congress as part of the Patient Protection and Affordable Care Act of 2010, is designed to promote medical research that could improve health and save lives.
 
The QTDP was intended specifically to provide incentive to smaller companies who are focusing on innovative therapeutic discoveries that show potential to produce new therapies that address areas of unmet medical need, and reduce long-term health care costs. The grant covers up to 50% of the cost of qualifying biomedical research and expenses for the tax years of 2009 and 2010, and is only available to firms with no more than 250 employees. In addition, awards took into consideration research that demonstrates the greatest potential to create and sustain high-quality, high-paying U.S. jobs and to advance U.S. competitiveness in life, biological and medical sciences.
 
"We are extremely pleased that our Thalagen™ project has been reviewed and assessed positively by the federal government, resulting in a grant at the maximum level available under the QTDP grant program" said Pat Girondi, EGT’s Chairman and Chief Executive Officer. "These funds and other sources continue to help support EGT’s ongoing research and development programs."
 
EGT said it will use the grant to develop Thalagen, a groundbreaking curative treatment for the deadly genetic blood disorder β-Thalassemia (also called Cooley’s Anemia). The company said it plans to launch the Phase 1 human clinical trials of Thalagen in Q1 2011.

About Errant Gene Therapeutics, LLC

Errant Gene Therapeutics™ (EGT) is a privately held biopharmaceutical company established in 2003. The company's mission is the development of treatments for life-threatening diseases with a special focus on rare diseases, commonly referred to as “orphan diseases.” EGT is developing its portfolio of products to correct the erroneous gene expression associated with many of these diseases. EGT therapies are currently being developed for hemoglobinopathies and selected cancers.

For more information about Errant Gene Therapeutics and its programs, visit www.errantgene.com or call (312) 441-1800 extension 11.

Waiting to hear more details from Pat on this, but I hope it can help keep Dr Sadelain and Sloan Kettering  on board.

This is amazing new - very encouraging.  I hope that more good news will follow.  Praying.

Sharmin

this is indeed a delightful news giving us new hope and reason to fight..........was so happy just by knowing about this and m waiting for a day when i would really hera the news of someone amongst our children getting cured by it
 ..............
                    Thank u for showing us the light...or a ray of hope other than the bone marrow

Time for an analogy.

There was a company that wanted to buy a race horse. They looked at two competing stables and even though the one stable offered a horse with the best possible breeding and training, this company chose a horse from another stable, even though there was some uncertainty about the horse's breeding and its long term viability as a race horse. They trained the horse and with a proper dose of drugs, this horse was able to win the Derby, as the other horse came in a strong second. However, after the race, the company looked at the horse and realized that this horse could never win another race, because it had never been a strong enough horse in the first place and had no long term viability. The company now looked at the horse that placed second with some degree of envy. This horse was in it for the long haul and would have many victories yet to come. So, realizing their own horse had won the only race it was going to win, the company went to the owner of the other horse and offered some big bucks if they would sell both horse and trainer to their company. The trainer, however, has been very happy working where he was and did not want to work for another and also, since the trainer had raised this horse since birth, he felt it was his baby and did not want someone else to come in and take over and tell him how he should train the horse. There also remained a good deal of distrust by this trainer for the trainer of the other horse, and he was very wary of this other company. But he was under contract to his stable and he would be obliged to do what they ruled. Meanwhile, as a result of the back and forth going on, the horse missed the next race, the Preakness Stakes. Now we have to wonder if this horse can possibly be ready for its next scheduled race at Belmont, as the owner and trainer can't agree on whether they should even race there, as the owner is now starry eyed over promised monies from the other company.

Folks, that is where we are right now with gene therapy. Stay tuned and see where this trainer and his horse end up and how much this delay will cost in the long term.

Story © A. Battaglia 2010

let's all pray together for gene therapy's success and availabilty in the very very near future!!

Pat Girondi and Andy Battaglia,

I am praying with all of my heart and soul for gene therapy to cure our children and our friends in the very near future. 

When this happens - I will have both of you to thank for your ongoing efforts and your commitment to us. 
Andy, thank you for your dedication and your courage to support and to be a voice for us.  We don't always understand what is going on - but you do and you have the courage and heart to ask the questions on our behalf. 

Pat, you are in the same situation that we are in - but you have pushed forward and made the changes that will help not only your own son - but all of us. 

You two are heros for us,

Thank you from the bottom of my heart,

Love Sharmin

i agree wth sharmin.......................we gonna pray about gene therapy daily in our prayers ................cos god does answer u

Thank you all for your support. I do myself no good in terms of my relations with thal organizations and some medical people by being so outspoken, but I am not one to be afraid to speak the truth, and when I am, I will know it is time to sit down and shut up.

Pat is a regular guy, very much like myself. Unassuming and not pretentious. I think these are good qualities but when it comes to negotiating with investment companies, this hasn't helped Pat. These people are used to meeting with other "suits" and a man like Pat may not meet their preconceived notions of what the head of a company should be. I think this has been a big factor on which group got financing for gene therapy, but now that company has had second thoughts, because any fool can see that if you want to succeed at perfecting this direction in gene therapy for thalassemia and do it so it is safe and effective, Michael Sadelain is going to have to be involved. (I would also like to make clear that I am not commenting on other directions in gene therapy, like what is being done by Dr Persons). So, right now when we should be seeing patients start on this trial, we are instead delayed yet again because monetary pressures have been put on our good doctor's employers, who have upped the per patient price and made unreasonable demands for money in advance. Meanwhile, another player has come into the picture and this could provide a much lower cost solution, but will the good doctor be allowed to participate by his employer? Hopefully, all will be made clear at this November 14 meeting.

Now, for my opinion on what should be done. Dr Sadelain's group has worked through Sloan Kettering for many years. It only makes sense to continue and do the trials there. However, SK is completely missing the big picture here as they try to latch on to some short term money. What would I do if I was in charge at SK? I feel like saying "duh" because I cannot fathom how this has slipped the grasp of the decision makers at SK.

Gene therapy is going to be huge and not just in terms of curing hemoglobinopathies. Once this process is developed and is viable, we will see it transferred to countless other genetic illnesses. This very fact alone, should be enough to convince SK to not only cooperate with the trials but to offer their services at no charge whatsoever. This short term investment is going to pay dividends thousands of times higher than any initial development. SK, how do you not see this? Do you wish to be left behind? Or do you want to be the leader in the next generation of curative medicine? SK, the ball is in your end of the court. Are you going to play or are you going to take your ball and go home?

I won't even address the moral issues of leaving patients hanging and the inevitable delay towards a cure that this is causing, because it has become quite evident that this is not a consideration by the decision makers. However, the eventual monetary bonanza that gene therapy will be should be plenty to convince the bean counters.

I am praying that this meeting goes well.  If this meeting goes our way, the way that all thalassemia patients and their families need it to go, then I believe that we will all have a much brighter future. 

Praying,

Sharmin

I expect a commitment from Dr Sadelain to continue working with Thalagen to perfect this process. We will know more after tomorrow, but I would be very surprised if this is not what we hear. NIH may become involved as a result.