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Bluebird Trials for Gene Therapy are Commencing

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Andy Battaglia:
Bluebird Bio is beginning trials for gene therapy in three locations in the US, and there is also a clinical site established in Australia, but they have not yet set up a trial, so no recruiting is taking place as of yet. They are already underway in France again and eventually expect to expand trials to Asia. I'd like to thank the people at Bluebird for supplying this information.


HGB-204 (“Northstar”) study:
·         Study website: http://www.northstarstudy.com
o   Frequently asked questions
o   Pre-screening tool to determine if patients meet basic eligibility requirements
·         ClinicalTrials.gov website: http://clinicaltrials.gov/show/NCT01745120
o   This shows that a clinical site in Oakland and a clinical site in Chicago are already actively recruiting
o   This also shows that a clinical site in Australia is active although not yet recruiting
o   This will be updated over time
·         No clinical data from this study will be presented at the EHA conference in Milan on June 14


HGB-205 study (based in France):
·         ClinicalTrials.gov website: http://clinicaltrials.gov/show/NCT02151526
·         Clinical data from the first two patients on this study will be presented at the EHA conference in Milan on June 14


Contacting bluebird bio:
·         For Beta Thalassemia patients or caregivers who are specifically interested in clinical studies:
o   Please feel free to contact: clinicaltrials@bluebirdbio.com
·         For Beta Thalassemia patients or caregivers who are *not* specifically interested in participating in clinical studies, but who would like to stay in touch with the company; who would like to receive updates about our progress; or who wish to receive information about patient-centric resources:
o   Please feel free to contact: patient.advocacy@bluebirdbio.com

Bostonian_04:
This is a great news..i feel hopeful that another 5 to 10 yrs there will be a cure for thal. Praying for success for Bluebird.

Andy, any news from Errant Gene trial that is ongoing at Sloan?

Andy Battaglia:
I know Sadelain presented new data in May and they will also be presenting in Chicago, but I have no information yet on what the new data showed. I feel that they could do a better job with public relations.

Canadian_Family:
bluebird bio to present initial clinical data from its HGB-205 Study in beta-thalassemia major patients at the 19th European Hematology Association Congress on June 14, 2014. For details, please see link below.

Summary of Abstract Data and Clinical Data to be Presented at EHA:
-Clinical data will be presented on two subjects with beta-thalassemia major (#1201 and #1202) transplanted with new lentiviral vector BB305 in the HGB-205 Study
-Vector copy number in the drug product for subjects 1201 and 1202 are 1.5 and 2.1 respectively; higher than the drug product vector copy numbers reported in the prior LG001 Study (0.3-0.6), which used the older lentiviral vector HPV569
-A detailed update on the clinical data and transfusion status of subjects 1201 and 1202 will be provided as part of the EHA presentation
 
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=1933355&highlight=

Sharmin:
Thank you for sharing this information Andy.  Thank you also for the update Canadian Family. 
I am praying that gene therapy will be safe and successful in treating thalassemia within the next few years. 

Sharmin

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