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Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials

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Pratik:

--- Quote from: Sharmin on February 21, 2013, 10:43:24 PM ---The timing of the release of this video is encouraging - as of yet however we do not know anything for certain about the progress of the gene therapy trials:

http://www.youtube.com/watch?v=gPPKvouhLvE&feature=youtu.be

--- End quote ---
That's certainly very encouraging.

ANI:
Really it was an amazing video. Vey Very encouraging . I just pray to God that the resuts come shortly and it becomes  equally encoraging for all of us.
God bless all of us
Bye

Canadian_Family:
Keeping you in the loop.

Update February 19, 2013

Dr. Boulad and Dr. Sadelain provided answers to your questions. They said the protocol is currently open until July 2014 and patients are still being enrolled. The timing of the results will depend on patient accrual numbers and the levels of gene expression that are seen. They hope these issues will both be solved by July 2014. If it moves forward, this therapy would be for all types of Beta-thalassemia major independent of the Beta-globin gene mutation. In order to start treating patients affected by Beta-thalassemia, all (or at least the majority) of patients would need to engraft with the gene and become transfusion independent without any major side effects. The next steps would be production of the vector in larger scale, FDA approval for the vector, and extending the trial to a Phase II study. Licensing the vector to a biotech company may be necessary to broaden access to this therapy, just as it is with any new medication, chemotherapy, or biologic agent.

As mentioned in the youtube video posted by Sharmin, it takes about $1million to prepare the vector for small number of patients. Licensing to a biotech compnay should bring the cost down.

Best of luck to everyone.

Sharmin:
Canadian Family,

Thank you kindly for the update.  It seems like we have taken the first steps in the right direction - and a lot is pending on gene expression in the patients and subsequently financing and manufacturing of the vector.  I hope that more patients will enroll in the study and be cured of thalassemia. 

I hope we begin to see excellent results from the first few patients!

Sharmin

Andy Battaglia:
I received this note from Pat G today.


--- Quote ---Dearest family,

The 2 treated patients continue to do fantastically. A third patient has already done the stem cell mobilization and will do the 'Gene Transplant' in July. The team will decide whether an increase in chemotherapy is necessary for the next 3 patients.

--- End quote ---

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