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Author Topic: Bluebird Bio gene therapy company  (Read 8828 times)
7assan
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« on: November 11, 2010, 03:00:32 AM »

bluebird bio is the leading gene therapy company for the development of breakthrough treatments for severe genetic disorders. 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. bluebird bio is conducting Phase I/II trials with its LentiGlobin™ product in Thalassemia, one of the 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. 
This is an exciting opportunity to join a privately held company working to identify novel therapies to treat rare genetic diseases.  You will work with leaders in field to discover and develop new therapies to treat debilitating diseases that have little or no treatment options available today. 

The successful candidate will participate in design and implementation of the establishment of viral vector producer lines, which includes molecular cloning, cell culture, titration, various immunological assays, flow cytometry and other related techniques.  Additionally, you will provide support in cell culture and gene transfection and detection, immunostaining, isotope labeling and detection, DNA/RNA purification and hybridization and qPCR.  You will be responsible for writing relevant technical reports and presenting research work as well as maintaining the virology and biosafety level II laboratory as a safe and organized working environment.


http://jobview.monster.com/Research-Associate-Vector-Biology-Job-Cambridge-MA-US-92035576.aspx
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« Reply #1 on: November 13, 2010, 11:03:12 AM »

i´m confused. successful gene therapy? 2 patıents cured?

ıs thıs true?
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Andy Battaglia
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« Reply #2 on: November 13, 2010, 11:33:27 AM »

This is the same company yet again, formerly Genetix, but with a brand new name. They have had that same one patient success after failing with the first patient. I would like to quote Dr Persons comments on this so we can maintain some perspective.

http://www.medscape.com/viewarticle/728656  (it may be necessary to create a free Medscape account to view the article).

Quote
"Whenever a hematologist or oncologist hears, in terms of your blood system, that one clone is that dominant, that frequent, that's usually associated with a bad thing. One [clone] shouldn't take over that much," said Derek A. Persons, MD, PhD, from the Department of Hematology and Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee, in a telephone interview with Medscape Medical News. Dr. Persons also commented on the study in a News & Views article in Nature.

"But you've got to remember, it's still only 7% of all the [blood] cells in the body. And the key thing is that it stopped growing, it plateaued, and it stayed stable," said Dr. Persons. "Cancer doesn't stay stable, so this guy doesn't have cancer. He's got his clone, and we don't know what's going to happen to it. Like I said at the ending of the [News & Views article], the only way we're going to really ever know is, we watch him."

I'm going back to what I asked previously. How do they expect the FDA to ever approve a process that involves a mutated gene? How long do we have to watch this patient before we know for sure it is safe? And would not using a normal beta globin gene be preferable?
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Andy

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7assan
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« Reply #3 on: November 13, 2010, 12:08:32 PM »

Yeah but i think this gene therapy company have already cure  patient with thalassemia and thery are doing same thing with other 10 patient and FDA have approved , so wht is porb with this company , finenac or wht ......or this comapny is fake wht is this compay story .....we want to know .......?
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Andy Battaglia
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« Reply #4 on: November 13, 2010, 12:14:17 PM »

This is the same story that has been posted here many times. They have managed tremendous media coverage from one case. But please keep in mind that others like Dr Sadelain and Dr Persons are both carrying on separate gene therapy research. Dr Sadelain will speak tomorrow and I am anxiously awaiting his message. His 10 patients who are ready to start trials are in no way related to anything Bluebird Bio is working on.
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Andy

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« Reply #5 on: November 14, 2010, 12:27:06 PM »

andy how do we watch Dr sadelaın ?
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Andy Battaglia
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« Reply #6 on: November 14, 2010, 01:05:31 PM »

I do not believe that this session was broadcast. Waiting to hear details.
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Andy

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« Reply #7 on: November 14, 2010, 04:55:39 PM »

Sadelain's talk was very good from what I have been told. He compared his process with the process used by the other company and made clear distinctions between the two. He is ready to start trials. The only question now is whether Sloan Kettering is ready to stop wasting time while another company waves dollars in their face. It would be much better if they told the other company to go back to Europe and stop trying to control the process that will really work. The bean counters at SK apparently can't see past the end of their noses.

I expect that we will soon see trials commence through a combination of efforts between the National Institute of Health and a begrudging Sloan Kettering. Sadelain is under contract to SK, but Sadelain owns the patent on the beta globin gene and Errant Gene owns the process. SK is doing all they can to take ownership away from Errant Gene, and this is unconscionable. It isn't supposed to be about who can get rich. It's supposed to be about curing people. The people at SK should be embarrassed that ANY delay is being caused by corporate greed, especially when they are being so short-sighted that they cannot see the future that gene therapy will bring to many various genetic ailments.

So, the news from this talk is good regarding the process of gene therapy but we are still waiting for a true commitment by SK to start trials. Sadelain will not wait much longer, so SK has to decide soon if they are going to lose much of this research to the NIH. It will move forward, so have no doubts about that.
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Andy

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« Reply #8 on: November 14, 2010, 09:54:08 PM »

Andy,

Thank you - first and foremost - for having the courage and heart to speak on our behalf in all circumstances.  Its a shame that pharmaceutical companies do not have our best interest at heart the way that you do. 

Second of all - we can all breath that this is going to move ahead.  I hope that there aren't any more delays.  I am so glad that Dr. Sadelain came through for us. 

Thank you for updating us Andy,

Sharmin
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7assan
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« Reply #9 on: November 15, 2010, 01:59:43 AM »

ThAnx andy 4 update , Good News But taking Long time , hope that will be in soon.





FaReS AL BaLoShi
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« Reply #10 on: March 23, 2011, 08:37:03 AM »

Bluebird Bio Awarded Up to $4.2 Million From the French Muscular Dystrophy Association for Continued Development of Gene Therapy for Beta-Thalassemia and Sickle Cell Anemia
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Friday, March 18, 2011
CAMBRIDGE, Mass. -- bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, announced that it has entered into an agreement with the French Muscular Dystrophy Association (AFM), a French non-profit entity, whereby the company will receive an initial amount of approximately $1.4 million in cash to support the development of LentiGlobin®, the company’s development-stage program for the treatment of beta-thalassemia and sickle cell anemia.

As part of the research agreement, bluebird bio has the option to draw upon an additional amount of up to $2.8 million in credit toward the manufacturing of cGMP clinical trial material at Généthon. In December 2010, bluebird bio entered into an agreement with Généthon designed to enable substantial advances in the existing manufacturing process of lentiviral vectors for the benefit of both partners.

bluebird bio’s LentiGlobin introduces a fully functional human beta-globin gene, under the control of the beta-globin promoter and locus control regions, into the patient's own hematopoietic stem cells. bluebird bio is currently conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell anemia. Based on clinical data published in the September 2010 issue of Nature, LentiGlobin therapy has shown the potential to eliminate the need for monthly blood transfusions in patients with beta-thalassemia, without the risk of graft-versus-host disease.

“This funding from the AFM will not only support our ongoing thalassemia and sickle cell clinical trial, but also signifies the beginning of an important collaboration with the AFM,” said Nick Leschly, president and chief executive officer of bluebird bio. “We are grateful for the AFM's commitment to the advancement of a treatment that has the potential to greatly improve the lives of patients.”

Beta-thalassemia is an inherited blood disorder that is named for defects in production of the beta-globin chain of hemoglobin, the protein in red blood cells that carries oxygen. Approximately 60,000 children are diagnosed with the disease each year throughout the world. Patients typically require monthly supportive red blood cell transfusions to treat their severe anemia for life. Sickle cell anemia is characterized by clotting of improperly shaped red blood cells, which leads to a wide variety of serious health problems including chronic pain and high risk for stroke. Sickle cell anemia affects millions throughout the world, including approximately 95,000 people in the United States.

About the AFM

The French Muscular Dystrophy Association (Association Française contre les Myopathies, AFM) was founded for the purpose of serving patients with genetic neuromuscular diseases (diseases that cause muscle wasting) and their parents. Thanks in great part to donations from France's annual Telethon (€95 million raised in 2009), the AFM has become a major player in biomedical research for rare diseases both in France and worldwide. It is currently funding 36 clinical trials for 30 different genetic diseases, including diseases of the eye, blood, brain, immune system and muscle. Thanks to its Généthon research lab, the AFM stands out through its unique ability to produce and test its own gene-based therapeutics.

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