Bluebird Bio

This video is very interesting - and tells us all that gene therapy is not just science fiction anymore - it has arrived. 

http://www.youtube.com/watch?v=5Zk9WqYD9nA

A few interesting things to note near the end of the video, if I'm not mistakened he says - myeloablation or myelosuppression depending on how well the procedure works.  Which tells me HOPEFULLY - that Bluebird will take Sloan Kettering's lead and lean toward myelosuppression whenever possible.  Also, his last comment was - hopefully soon they will be able to do gene editing - which makes me think of Crispr and nanotechnology. 

The future is bright - patients - maintain good hemoglobins and keep your iron low - within the normal range if possible!!  Be in the best shape possible to take advantage of all of the promising treatments awaiting you. 

bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that bluebird bio and its clinical investigator plan to provide an oral presentation on the HGB-205 Study in beta-thalassemia major patients at the 19th Annual Congress of the European Hematology Association (EHA) in Milan, Italy from June 12-15, 2014.

Abstract Title: Outcomes of gene therapy for beta-thalassemia major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral beta globin vector.

Lead Author: Marina Cavazzana, M.D.

Session Title: Simultaneous Sessions & EHA Advocacy Sessions

Presentation Date/Time: Saturday, June 14 from 4:15 pm CET to 4:30 pm CET (10:15 am EDT to 10:30 am EDT)

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://www.streetinsider.com/Corporate+News/Bluebird+Bio+(BLUE)+to+Present+Clinical+Data+From+its+HGB-205+Study/9513611.html

Thanks for sharing.

This puts pressure on Memorial Sloan Kettering Cancer Center to come with their study results. However, don't forget that Blue is listed on NASDAQ (companies sometime do these gimmicks to improve the share prices).

Lets hope they have substantial news for the thal community.

thank you for sharing. 

I agree CF - I'm cautiously optimistic...